aripiprazole lauroxil

mrdef: prodrug of aripiprazole, following intramuscular injection is converted by enzyme-mediated hydrolysis to N-hydroxymethyl aripiprazole which is then hydrolyzed to aripiprazole

description: A dodecanoate ester obtained by formal condensation of the carboxy group of dodecanoic acid with the hydroxy group of 7-{4-[4-(2,3-dichlorophenyl)piperazin-1-yl]butoxy}-2-oxo-3,4-dihydroquinolin-1(2H)-yl]methanol. A prodrug for aripiprazole, it is used for treatment of schizophrenia.


terodiline

mrdef: Terodiline relaxes the smooth muscle and used to reduce bladder tone in treatment of urinary frequency and incontinence. Muscle relaxation caused by terodiline, is probably due to its anticholinergic and calcium antagonist activity


mandelic acid

A 2-hydroxy monocarboxylic acid that is acetic acid in which two of the methyl hydrogens are substituted by phenyl and hydroxyl groups.


glymidine

mrdef: was MH 1975-92 (see under SULFONAMIDES 1975-90); GLIDIAZINE & GLYCODIAZINE were see GLYMIDINE 1975-92; use SULFONAMIDES to search GLYMIDINE 1975-92; a sulfonamide hypoglycemic agent which stimulates insulin secretion

description: A sulfonamide that is N-(pyrimidin-2-yl)benzenesulfonamide which is substituted at position 5 of the pyrimidine ring by a 2-methoxyethoxy group. It is a hypoglycemic drug used for the treatment of diabetes mellitus.


idarucizumab

mrdef: an anticoagulant reversal agent that targets dabigatran


name: Drug-induced coagulation inhibitor disorder

category: biolink:Disease biolink:DiseaseOrPhenotypicFeature biolink:BiologicalEntity biolink:ThingWithTaxon biolink:NamedThing


insulin glulisine

mrdef: Insulin glulisine [rDNA origin] is a rapid-acting human insulin analog used to lower blood glucose. Insulin glulisine is produced by recombinant DNA technology utilizing a non-pathogenic laboratory strain of Escherichia coli (K12). Insulin glulisine differs from human insulin in that the amino acid asparagine at position B3 is replaced by lysine and the lysine in position B29 is replaced by glutamic acid.


mepolizumab

mrdef: Mepolizumab is an interleukin-5 antagonist (IgG1 kappa). IL-5 is the major cytokine responsible for the growth and differentiation, recruitment, activation, and survival of eosinophils. Mepolizumab binds to IL-5 with a dissociation constant of 100 pM, inhibiting the bioactivity of IL-5 by blocking its binding to the alpha chain of the IL-5 receptor complex expressed on the eosinophil cell surface.


vinpocetine

mrdef: whole issue of Arzneim Forsch (23 articles) discuss this drug; Arzneim Forsch 26(10a);1976; RN given refers to parent cpd with unspecified isomeric designation


osimertinib

mrdef: Osimertinib is a kinase inhibitor of the epidermal growth factor receptor (EGFR), which binds irreversibly to certain mutant forms of EGFR (T790M, L858R, and exon 19 deletion) at approximately 9-fold lower concentrations than wild-type. In cultured cells and animal tumor implantation models, osimertinib exhibited anti-tumor activity against NSCLC lines harboring EGFR-mutations (T790M/L858R, L858R, T790M/exon 19 deletion, and exon 19 deletion) and, to a lesser extent, wild-type EGFR amplifications.

status: OFP

description: A member of the class of aminopyrimidines that is 4-(1-methylindol-3-yl)pyrimidin-2-amine in which one of the amino hydrogens is replaced by a 2-methoxy-4-[2-(dimethylamino)ethyl](methyl)amino-5-acrylamidophenyl group. Used (as the mesylate salt) for treatment of EGFR T790M mutation positive non-small cell lung cancer.


hexamidine

A polyether that is the bis(4-guanidinophenyl) ether of hexane-1,6-diol.


Acanthamoeba keratitis

description: Keratitis due to infection by acanthamoeba; it is usually associated with soft contact lens wear, particularly overnight wear.


bencyclane

mrdef: A vasodilator agent found to be effective in a variety of peripheral circulation disorders. It has various other potentially useful pharmacological effects. Its mechanism may involve block of calcium channels.


clomethiazole

mrdef: A sedative and anticonvulsant often used in the treatment of alcohol withdrawal. Chlormethiazole has also been proposed as a neuroprotective agent. The mechanism of its therapeutic activity is not entirely clear, but it does potentiate GAMMA-AMINOBUTYRIC ACID receptors response and it may also affect glycine receptors.


dichloroacetic acid

mrdef: A derivative of ACETIC ACID that contains two CHLORINE atoms attached to its methyl group.

smiles: OC(=O)C(Cl)Cl

description: An organochlorine compound comprising acetic acid carrying two chloro substituents at the 2-position. It occurs in nature in seaweed, Asparagopsis taxiformis.


patiromer calcium

mrdef: binds potassium ions in the gastrointestinal tract to lower serum potassium levels


alcoholic hepatitis

description: Acute hepatitis resulting from ingestion of alcohol.


leiomyosarcoma

description: An uncommon, aggressive malignant smooth muscle neoplasm, usually occurring in post-menopausal women. It is characterized by a proliferation of neoplastic spindle cells. Morphologic variants include epithelioid, granular cell, inflammatory and myxoid leimyosarcomas.


ferric maltol

mrdef: potential use as an oral therapy for iron deficiency anemia


selexipag

mrdef: an oral prostacyclin receptor (IP receptor) agonist that is structurally distinct from prostacyclin, indicated for the treatment of pulmonary arterial hypertension, selexipag is hydrolyzed by carboxylesterase 1 to yield its active metabolite, which is approximately 37-fold as potent as selexipag, selexipag and the active metabolite are selective for the IP receptor versus other prostanoid receptors (EP1-4, DP, FP and TP)

status: ONP

description: A member of the class of pyrazines that is N-(methanesulfonyl)-2-{4-[(propan-2-yl)(pyrazin-2-yl)amino]butoxy}acetamide carrying two additional phenyl substituents at positions 5 and 6 on the pyrazine ring. An orphan drug used for the treatment of pulmonary arterial hypertension. It is a prodrug for ACT-333679 (the free carboxylic acid).


primary myelofibrosis

description: Myelofibrosis with myeloid metaplasia is a myeloproliferative disease with annual incidence of approximately 1 case per 100,000 individuals and age at diagnosis around 60 (an increased prevalence is noted in Ashkenazi Jews). Clinical manifestations depend on the type of blood cell affected and may include anemia, pallor, splenomegaly, hypermetabolic state, petechiae, ecchymosis, bleeding, lymphadenopathy, hepatomegaly, portal hypertension.


lobeglitazone

mrdef: putative antidiabetic agent for type 2 diabetes


Community acquired pneumonia

description: Pneumonia that is not acquired in a hospital or long-term care facility setting.


viloxazine

mrdef: A morpholine derivative used as an antidepressant. It is similar in action to IMIPRAMINE. The mechanism of action of viloxazine in the treatment of ADHD is unclear; however, it is thought to be through inhibiting the reuptake of norepinephrine.


proglumetacin

A carboxylic ester obtained by formal condensation of the carboxy group of indometacin with the hydroxy group of 3-[4-(2-hydroxyethyl)piperazin-1-yl]propyl N(2)-benzoyl-N,N-dipropyl-alpha-glutaminate. Used (as its dimaleate salt) to control pain and inflammation associated with musculoskeletal and joint disorders. Following oral administration, it is metabolised to indometacin and proglumide, a drug with antisecretory effects that helps prevent injury to the stomach lining.


belatacept

mrdef: Belatacept, a selective T-cell (lymphocyte) costimulation blocker, binds to CD80 and CD86 on antigen-presenting cells thereby blocking CD28 mediated costimulation of T lymphocytes. In vitro, belatacept inhibits T lymphocyte proliferation and the production of the cytokines interleukin-2, interferon-gamma, interleukin-4, and TNF-alfa. Activated T lymphocytes are the predominant mediators of immunologic rejection


glucose

mrdef: A primary source of energy for living organisms. It is naturally occurring and is found in fruits and other parts of plants in its free state. It is used therapeutically in fluid and nutrient replacement.

description: D-Glucopyranose with beta configuration at the anomeric centre.


defibrotide

mrdef: Single-stranded polydeoxyribonucleotide extracted from mammalian organs and used in the treatment of HEPATIC VENO-OCCLUSIVE DISEASE in patients with kidney or lung abnormalities following HEMATOPOIETIC STEM CELL TRANSPLANTATION. The compound is a sodium salt.


hepatic veno-occlusive disease

description: Hepatic veno-occlusive disease (hepatic VOD) is a condition resulting from toxic injury to the hepatic sinusoidal capillaries that leads to obstruction of the small hepatic veins.


obiltoxaximab

mrdef: A monoclonal antibody that neutralizes toxins produced by BACILLUS ANTHRACIS. It is used to treat inhalational ANTHRAX.


thallous chloride Tl-201

cd_formula: ClTl

mrdef: non-pyrogenic diagnostic radiopharmaceutical for intravenous administration


Myocardial imaging

category: biolink:Procedure biolink:ActivityAndBehavior biolink:NamedThing biolink:Occurrent biolink:PhysicalEssenceOrOccurrent


reslizumab

mrdef: Reslizumab is an interleukin-5 antagonist (IgG4, kappa). IL-5 is the major cytokine responsible for the growth and differentiation, recruitment, activation, and survival of eosinophils. Reslizumab binds to IL-5 with a dissociation constant of 81 pM, inhibiting the bioactivity of IL-5 by blocking its binding to the alpha chain of the IL-5 receptor complex expressed on the eosinophil surface. Inflammation is an important component in the pathogenesis of asthma. Multiple cell types (e.g., mast cells, eosinophils, neutrophils, macrophages, lymphocytes) and mediators (e.g., histamine, eicosanoids, leukotrienes, cytokines) are involved in inflammation. Reslizumab, by inhibiting IL-5 signaling, reduces the production and survival of eosinophils; however, the mechanism of reslizumab action in asthma has not been definitively established.


octocog alfa

mrdef: Octocog alfa (human coagulation factor VIII), is a substance that helps the blood to clot. Patients with haemophilia A lack factor VIII, and this causes blood clotting problems, such as bleeding in the joints, muscles or internal organs. Advate is used to correct the factor VIII deficiency by replacing the missing factor VIII, giving temporary control of the bleeding disorder. Octocog alfa is made by a method known as ‘recombinant DNA technology’, it is made by cells into which a gene (DNA) has been introduced that makes them able to produce it.


galsulfase

mrdef: a recombinant form of N-acetylgalactosamine-4-sulfatase


mucopolysaccharidosis type 6

description: Mucopolysaccharidosis type 6 (MPS 6) is a lysosomal storage disease with progressive multisystem involvement, associated with a deficiency of arylsulfatase B (ASB) leading to the accumulation of dermatan sulfate.


sutilains

mrdef: used in treatment of burns; derived from filtration of cultures of a strain of Bacillus subtilis


acrisorcin

mrdef: A topical anti-fungal agent. Acrisorcin is comprised of two active ingredients, 9-aminoacridine and 4-hexylresorcinol, but its clinical use has been discontinued.


rilonacept

mrdef: an anti-arthritis agent derived from IL-1R and IL-1RAcP


nonoxinol

mrdef: Nonionic surfactant mixtures varying in the number of repeating ethoxy (oxy-1,2-ethanediyl) groups. They are used as detergents, emulsifiers, wetting agents, defoaming agents, etc. Nonoxynol-9, the compound with 9 repeating ethoxy groups, is a spermatocide, formulated primarily as a component of vaginal foams and creams.

description: A tergitol polymer consisting of nonylbenzene with a nine-membered poly(ethylene glycol) moiety attached at position 4.


beractant

mrdef: a bovine lung extract that contains mostly phospholipids; modified by the addition of dipalmitoylphosphatidylcholine, palmitic acid, and tripalmitin


urokinase

mrdef: A proteolytic enzyme that converts PLASMINOGEN to FIBRINOLYSIN where the preferential cleavage is between ARGININE and VALINE. It was isolated originally from human URINE, but is found in most tissues of most VERTEBRATES.


migalastat

mrdef: Migalastat is a pharmacological chaperone that is designed to selectively and reversibly bind with high affinity to the active sites of certain mutant forms of alpha-Gal A, the genotypes of which are referred to as amenable mutations. Migalastat binding stabilizes these mutant forms of alpha-Gal A in the endoplasmic reticulum and facilitates their proper trafficking to lysosomes where dissociation of migalastat restores alpha-Gal A activity, leading to the catabolism of GL-3 and related substrates.


hemin

It is used as a prescription medication to relieve repeated attacks of acute intermittent porphyria related to the menstrual cycle in affected women.


technetium Tc 99m medronate

mrdef: A radiopharmaceutical containing methylene diphosphonate (medronate; MDP) complexed with the gamma-emitting radionuclide technetium Tc 99m with radioisotopic activity and hydroxyapatite affinity. Upon intravenous administration, skeletal uptake of technetium Tc 99m methylene diphosphonate occurs as a function of skeletal blood flow and osteogenic activity. The MDP moiety of this agent has affinity for hydroxyapatite crystals in bone with abnormal accumulation at sites with increased osteoid mineralization; labeling of MDP with Tc 99m allows scintigraphic imaging of areas of abnormal osteogenesis associated with malignant bone lesions.


sucroferric oxyhydroxide

mrdef: an oral phosphate binder for treatment of hyperphosphatemia

description: An iron oxide of nanoparticulate size and poor crystallinity. It is a mineral commonly found in soil and freshwater environments and forms a core structural component in the Fe-buffering ferritin protein. It is also used in the sequestration of heavy element contaminants, and is a precursor of haematite and goethite.


technetium (99mTc) sestamibi

mrdef: A technetium imaging agent used to reveal blood-starved cardiac tissue during a heart attack.


omega-3-carboxylic acids

mrdef: Fish oil-derived free fatty acids including multiple omega-3 fatty acids, eicosapentaenoic acid [EPA] and docosahexaenoic acid [DHA] being the most abundant. The mechanism of action of omega-3-carboxylic acids is not completely understood. Potential mechanisms of action include inhibition of acyl-CoA:1,2-diacylglycerol acyltransferase, increased mitochondrial and peroxisomal beta-oxidation in the liver, decreased lipogenesis in the liver, and increased plasma lipoprotein lipase activity. Omega-3-carboxylic acids may reduce the synthesis of triglycerides in the liver because EPA and DHA are poor substrates for the enzymes responsible for TG synthesis, and EPA and DHA inhibit esterification of other fatty acids.


ferric carboxymaltose

mrdef: effective for treatment of postpartum anemia


diloxanide furoate

A carboxylic ester resulting from the formal condensation of the carboxy group of furan-2-carboxylic acid with the hydroxy group of 2,2-dichloro-N-(4-hydroxyphenyl)-N-methylacetamide. It is a drug used for the treatment of asymptomatic amebiasis.


alglucerase

mrdef: modified form of human placental glucocerebrosidase; oligosaccharide units of glucocerebrosidase are sequentially deglycosylated to yield a mannose-terminated preparation which is targeted to the mannose lectin on the macrophage plasma membrane


atezolizumab

mrdef: A monoclonal antibody that targets programmed death-ligand 1 (CD274 ANTIGEN) and is used to treat urothelial carcinoma, the most common type of bladder cancer.


conestat alfa

mrdef: recombinant human complement component 1 (C1) esterase inhibitor (rhC1INH), is an analogue of human C1INH and is obtained from the milk of rabbits expressing the gene encoding for human C1INH, the amino acid sequence of conestat alfa is identical to that of endogenous C1INH


hereditary angioedema type 1

description: Hereditary angioedema type 1 (HAE 1) is a form of hereditary angioedema characterized by acute edema in subcutaneous tissues, viscera and/or the upper airway.


fluciclovine (18F)

mrdef: a radiotracer containing a synthetic amino acid analogue of L-leucine radiolabeled with fluorine F 18 with diagnostic imaging use

description: A member of the class of cyclobutanes that is cyclobutane which carries a carboxy, amino and ((18)F)fluoro groups at positions 1, 1 and 3, respectively (the 1r,3r-stereoisomer). It is a positron emission tomography (PET) radiotracer for visualizing prostate cancer.


velpatasvir

mrdef: pan-genotypic NS5A inhibitor for the treatment of chronic genotype 1-6 hepatitis C virus (HCV) infection

description: A complex organic heteropentacyclic compound that is a hepatitis C virus nonstructural protein 5A inhibitor used in combination with sofosbuvir (under the brand name Epclusa) for treatment of patients with chronic hepatitis C of all six major genotypes.


terguride

mrdef: pronounced antifertility agent in rats; lactation suppressor in other species; serotonin antagonist; RN given refers to parent cpd; structure


Gallium Ga 68 dotatate

mrdef: A radioactive diagnostic agent used for POSITRON EMISSION TOMOGRAPHY (PET) imaging of SOMATOSTATIN RECEPTOR positive neuroendocrine tumors and malignant abdominal paraganglioma.


carbenoxolone

mrdef: An agent derived from licorice root. It is used for the treatment of digestive tract ulcers, especially in the stomach. Antidiuretic side effects are frequent, but otherwise the drug is low in toxicity.


mebhydrolin

mrdef: see also record for mebhydroline, RN: 524-81-2


tilidine

mrdef: An opioid analgesic used similarly to MORPHINE in the control of moderate to severe pain. (From Martindale, The Extra Pharmacopoeia, 30th ed, p1097)

description: An amino acid ester that is the ethyl ester of cyclohex-3-ene-1-carboxylic acid in which the cyclohexane ring is substituted at positions 1 and 2 by phenyl and dimethylamino groups, respectively. It has four possible diastereoisomers; the opioid analgesic drug tilidine is a racemate comprising the two trans diastereoisomers.


neltenexine

mrdef: elastase inhibitor; may be useful for preventing pulmonary emphysema


mosapramine

A racemate comprising equimolar amounts of (R)- and (S)-mosapramine. It is a second-generation antipsychotic used for the treatment of schizophrenia.


obeticholic acid

mrdef: obeticholic acid is an agonist for FXR, a nuclear receptor expressed in the liver and intestine

description: A dihydroxy-5beta-cholanic acid that is chenodeoxycholic acid carrying an additional ethyl substituent at the 6alpha-position. A semi-synthetic bile acid which acts as a farnesoid X receptor agonist and is used for treatment of primary biliary cholangitis.


spiperone

mrdef: A spiro butyrophenone analog similar to HALOPERIDOL and other related compounds. It has been recommended in the treatment of SCHIZOPHRENIA.

description: An azaspiro compound that is 1,3,8-triazaspiro[4.5]decane which is substituted at positions 1, 4, and 8 by phenyl, oxo, and 4-(p-fluorophenyl)-4-oxobutyl groups, respectively.


lonidamine

A member of the class of indazoles that is 1H-indazole that is substituted at positions 1 and 3 by 2,4-dichlorobenzyl and carboxy groups, respectively.


azidocillin

mrdef: antibiotic similir to ampicillin; minor descriptor (75-86); on line & INDEX MEDICUS search PENICILLIN G/AA (75-86); RN given refers to (2S-(2alpha,5alpha,6beta(S*))-isomer)


butriptyline

mrdef: a tricyclic antianxiety and antidepressive agent similar to, but with less side effects than amitriptyline; minor descriptor (75-86); on line & INDEX MEDICUS search DIBENZOCYCLOHEPTENES (75-86); RN given refers to parent cpd


scopolamine butylbromide

mrdef: Antimuscarinic quaternary ammonium derivative of scopolamine used to treat cramps in gastrointestinal, urinary, uterine, and biliary tracts, and to facilitate radiologic visualization of the gastrointestinal tract.

description: A carboxylic ester resulting from the formal condensation of the carboxy group of (2S)-3-hydroxy-2-phenylpropanoic acid with the hydroxy group of (2R,4S,5S,7s)-9-butyl-7-hydroxy-9-methyl-3-oxa-9-azoniatricyclo[3.3.1.0(2,4)]nonane.


ethanol

mrdef: A clear, colorless liquid rapidly absorbed from the gastrointestinal tract and distributed throughout the body. It has bactericidal activity and is used often as a topical disinfectant. It is widely used as a solvent and preservative in pharmaceutical preparations as well as serving as the primary ingredient in ALCOHOLIC BEVERAGES.

smiles: CCO

description: A primary alcohol that is ethane in which one of the hydrogens is substituted by a hydroxy group.


methylpentynol

mrdef: a tertiary hexanol with hypnotic/sedative and anticonvulsant effects, was used for the treatment of insomnia

smiles: CCC(C)(O)C#C


choline

mrdef: A basic constituent of lecithin that is found in many plants and animal organs. It is important as a precursor of acetylcholine, as a methyl donor in various metabolic processes, and in lipid metabolism.

smiles: C[N+](C)(C)CCO

description: A choline that is the parent compound of the cholines class, consisting of ethanolamine having three methyl substituents attached to the amino function.


betazole

mrdef: A histamine H2 agonist used clinically to test gastric secretory function.

status: OFM

description: Pyrazole in which a hydrogen adjacent to one of the nitrogen atoms is substituted by a 2-aminoethyl group. It is a histamine H2-receptor agonist used clinically to test gastric secretory function.


orazamide

An aminoimidazole in which the amino group is at C-5 with a carboxamido group at C-4.


meptazinol

mrdef: A narcotic antagonist with analgesic properties. It is used for the control of moderate to severe pain.


epoetin beta

mrdef: A recombinant therapeutic agent that is chemically identical to or similar to the endogenous cytokine human erythropoietin (EPO). Produced primarily by cells of the peritubular capillary endothelium of the kidney in response to hypoxia, circulating EPO binds to EPO receptors on the surface of committed erythroid progenitors in the bone marrow resulting in their replication and maturation into functional erythrocytes.


pancrelipase

mrdef: A standardized enzyme concentrate containing the pancreatic enzymes, lipase, protease and amylase used in enzyme substitution therapy. Lipase, protease and amylase break down fat, protein, and starches, respectively in the small intestine, thereby promoting digestion. Pancrelipase is used to reduce malabsorption when the pancreas is unable to secrete sufficient amounts of these enzymes.


nadroparin calcium

mrdef: A low molecular weight heparin (LMWH) composed of a heterogeneous mixture of sulfated polysaccharide glycosaminoglycan chains obtained by depolymerisation of porcine mucosal sodium heparin, extraction/purification and conversion to the calcium salt. Nadroparin binds to antithrombin III (ATIII) and inhibits the activity of activated factor X (factor Xa), thereby inhibiting the final common pathway of the coagulation cascade and preventing the formation of a cross-linked fibrin clot


peginterferon alfa-2b

mrdef: A covalent conjugate of recombinant interferon alpha, subtype 2b, and polyethylene glycol (PEG), used as an antiviral and antineoplastic agent. The biological activity of this agent is derived from its interferon alpha-2b protein moiety. Interferons alfa bind to specific cell-surface receptors, leading to the transcription and translation of genes whose protein products mediate antiviral, antiproliferative, anticancer, and immune-modulating effects. The PEG moiety lowers the clearance of interferon alpha-2b, thereby extending the duration of its therapeutic effects, but may also reduce the interferon-mediated stimulation of an immune response


name: methoxy polyethylene glycol-epoetin beta

mrdef: A pegylated form of recombinant human erythropoietin, a glycosylated protein naturally produced in the kidney that stimulates erythrocyte production in the bone marrow. Methoxypolyethylene glycol epoetin beta may reverse anemias induced by cancer therapy


moroctocog alfa

mrdef: Human coagulation factor VIII produced by recombinant DNA technology in Chinese hamster ovary (CHO) cells. Moroctocog alfa is a glycoprotein with 1438 amino acids with a sequence that is comparable to the 90 + 80 kDa form of factor VIII (i.e. B-domain deleted) and similar post-translational modifications to those of the plasma-derived molecule


lenograstim

mrdef: A glycosylated form of a recombinant therapeutic agent which is chemically identical to or similar to an endogenous human granulocyte colony-stimulating factor (G-CSF). Produced endogenously by monocytes, fibroblasts, and endothelial cells, G-CSF binds to and activates specific cell surface receptors, stimulating neutrophil progenitor proliferation and differentiation and selected neutrophil functions.


eldecalcitol

mrdef: an analog of vitamin D for the treatment of osteoporosis

description: A hydroxycalciol that is calcitriol with a 3-hydroxypropoxy group at position 2.


epoetin zeta

mrdef: a copy of human erythropoietin and works in exactly the same way as the natural hormone to stimulate red blood cell production


nimetazepam

A nitrazepam which is substituted at positions 1 by a methyl group. It is used as an anticonvulsant and as a hypnotic for the short-term management of insomnia.


alfacalcidol

A member of the class of D3 vitamins that is calciol in which the hydrogen at the 1alpha position is replaced by a hydroxy group. It is an active metabolite of cholecalciferol, which performs important functions in regulation of the calcium balance and the bone metabolism.


alipogene tiparvovec

mrdef: gene therapy product, contains the human lipoprotein lipase (LPL) gene variant LPLS447X in a vector comprised of a protein shell derived from adeno-associated virus serotype 1 (AAV1), the Cytomegalovirus (CMV) promoter, a woodchuck hepatitis virus posttranscriptional regulatory element and AAV2 derived inverted terminal repeats


familial lipoprotein lipase deficiency

description: Familial lipoprotein lipase deficiency is a rare genetic disorder is which a person lacks the enzyme lipoprotein lipase, a protein needed to break down fat molecules. Deficiency of this enzyme prevents affected individuals from properly digesting certain fats. This results in the accumulation of fatty droplets called chylomicrons in the blood and an increase in the blood concentration of triglycerides. Symptoms include episodes of abdominal pain, recurrent inflammation of the pancreas (pancreatitis), abnormal enlargement of the liver and/or spleen (hepatosplenomegaly), and the development of skin lesions known as erruptive xanthomas. Familial lipoprotein lipase deficiency is caused by changes (mutations) in the LPL gene. It is inherited in an autosomal recessive pattern. Treatment aims to control symptoms and blood triglyceride levels with a very low-fat diet. Treatment for individual symptoms (i.e. pancreatitis) involves following established treatment guidelines.


clopenthixol

mrdef: A thioxanthene with therapeutic actions similar to the phenothiazine antipsychotics. It is an antagonist at D1 and D2 dopamine receptors.

description: A thioxanthene derivative having a chloro substituent at the 2-position and an alkylidene group at the 10-position with undefined double bond stereochemistry.


egualen

mrdef: a stable azulene derivative, antiulcer agent

description: A member of the class of azulenes carrying sulfo, ethyl and isopropyl substituents at positions 1, 3 and 7 respectively. Used (in the form of its sodium salt) for treatment of gastric ulcers.


calcium dobesilate

mrdef: A drug used to reduce hemorrhage in diabetic retinopathy.


cyclophosphamide

mrdef: Precursor of an alkylating nitrogen mustard antineoplastic and immunosuppressive agent that must be activated in the LIVER to form the active aldophosphamide. It has been used in the treatment of LYMPHOMA and LEUKEMIA. Its side effect, ALOPECIA, has been used for defleecing sheep. Cyclophosphamide may also cause sterility, birth defects, mutations, and cancer.

description: A phosphorodiamide that is 1,3,2-oxazaphosphinan-2-amine 2-oxide substituted by two 2-chloroethyl groups at the amino nitrogen atom.


retinoblastoma

description: A malignant tumor that originates in the nuclear layer of the retina. As the most common primary tumor of the eye in children, retinoblastoma is still relatively uncommon, accounting for only 1% of all malignant tumors in pediatric patients. Approximately 95% of cases are diagnosed before age 5. These tumors may be multifocal, bilateral, congenital, inherited, or acquired. Seventy-five percent of retinoblastomas are unilateral; 60% occur sporadically. A predisposition to retinoblastoma has been associated with 13q14 cytogenetic abnormalities. Patients with the inherited form also appear to be at increased risk for secondary nonocular malignancies such as osteosarcoma, malignant fibrous histiocytoma, and fibrosarcoma.


vindesine

mrdef: Vinblastine derivative with antineoplastic activity against CANCER. Major side effects are myelosuppression and neurotoxicity. Vindesine is used extensively in chemotherapy protocols (ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS).


dimethicone

mrdef: A poly(dimethylsiloxane) which is a polymer of 200-350 units of dimethylsiloxane, along with added silica gel. It is used as an antiflatulent, surfactant, and ointment base.

description: A polydimethylsiloxane macromolecule, composed of dimethylsiloxane repeating units.


bendazol

mrdef: has a hypotensive, vasodilator, antispasmodic action


von Willebrand factor

mrdef: a purified recombinant von Willebrand factor (rVWF) expressed in Chinese Hamster Ovary (CHO) cells, acts to promote hemostasis by mediating platelet adhesion to damaged vascular sub-endothelial matrix and platelet aggregation and as a carrier protein for factor VIII, protecting it from rapid proteolysis


fenproporex

mrdef: an anorectic drug used in the treatment of obesity


mizolastine

mrdef: a long-acting H1-antihistamine indicated for the symptomatic relief of seasonal allergic rhinoconjunctivitis (hay fever), perennial allergic rhinoconjunctivitis and urticaria


gadodiamide

mrdef: for intravenous administration with MRI in adult patients for contrast enhancement of CNS lesions

description: The hydrate of gadodiamide.


taurine

mrdef: A conditionally essential nutrient, important during mammalian development. It is present in milk but is isolated mostly from ox bile and strongly conjugates bile acids.

smiles: NCCS(O)(=O)=O

description: An amino sulfonic acid that is the 2-amino derivative of ethanesulfonic acid. It is a naturally occurring amino acid derived from  methionine and cysteine metabolism. An abundant component of fish- and meat-based foods, it has been used as an oral supplement in the treatment of disorders such as cystic fibrosis and hypertension.


N-Acetyltyrosine

An N-acetyltyrosine in which the chiral centre has L configuration.


phosphoric acid

mrdef: concise etchant is 37% H3PO4

smiles: OP(O)(O)=O

description: A phosphorus oxoacid that consists of one oxo and three hydroxy groups joined covalently to a central phosphorus atom.


AMMONIA N-13

A (13)N-modified compound that is ammonia that has a (13)N isotope as the nitrogen atom. (13)N decays with a half-life of ten minutes to (13)C, emitting a positron. Used in diagnostic Positron Emission Tomography (PET) imaging.


peginterferon alfa-2a

mrdef: interferon alfa‑2a attached to polyethylene glycol


omoconazole

mrdef: an imidazole antifungal which inhibits the cytochrome P450-dependent enzymes, resulting in impairment of ergosterol synthesis in fungal cell membranes


dermatophytosis

description: A common fungal infection of the stratum corneum of the skin, hair, or nails by a dermatophyte. It is characterized by itching, inflammation, redness of the skin, small papular vesicles, central clearing, fissures, scaling, and/or hair loss in the affected area.


nandrolone decanoate

mrdef: Decanoic acid ester of nandrolone that is used as an anabolic agent to prevent or treat WASTING SYNDROME associated with severe chronic illness or HIV infection (HIV WASTING SYNDROME). It may also be used in the treatment of POSTMENOPAUSAL OSTEOPOROSIS.


hydrogen peroxide

mrdef: A strong oxidizing agent used in aqueous solution as a ripening agent, bleach, and topical anti-infective. It is relatively unstable and solutions deteriorate over time unless stabilized by the addition of acetanilide or similar organic materials.

smiles: OO

description: An inorganic peroxide consisting of two hydroxy groups joined by a covalent oxygen-oxygen single bond.


vinyl ether

mrdef: a clear, nearly colorless, volatile liquid which was used as an inhalation anesthetic

smiles: C=COC=C


ebastine

mrdef: non-sedating second generation antihistamine which is used for allergic disorders


isopropanol

mrdef: An isomer of 1-PROPANOL. It is a colorless liquid having disinfectant properties. It is used in the manufacture of acetone and its derivatives and as a solvent. Topically, it is used as an antiseptic.

smiles: CC(C)O

description: A secondary alcohol that is propane in which one of the hydrogens attached to the central carbon is substituted by a hydroxy group.


sodium gluconate

An organic sodium salt having D-gluconate as the counterion.


cycrimine

description: A member of the class of piperidines that is 3-(piperidin-1-yl)propan-1-ol in which one of the hydrogen atoms at the 1-position is substituted by cyclopentyl, and the other is substituted by phenyl. A central anticholinergic, it is used as its hydrochloride salt in the management and treatment of Parkinson's disease.


mycetoma

description: Mycetomas are subcutaneous inflammatory pseudotumors containing fungal or actinomycetic (bacteria with branched filaments) granules or grains.


mrdef: a murine monoclonal antibody to the CD3 antigen of human T cells which functions as an immunosuppressant


eptacog alfa (activated)

mrdef: Eptacog alfa (activated) is recombinant coagulation factor VIIa (rFVIIa) with a molecular mass of approximately 50,000 Daltons produced in baby hamster kidney cells (BHK Cells) by recombinant DNA technology


Glanzmann thrombasthenia 1

description: A bleeding syndrome characterized by spontaneous mucocutaneous bleeding and an exaggerated response to trauma due to a constitutional thrombocytopenia


factor VII deficiency

description: A coagulation disorder characterized by the partial or complete absence of factor VII activity in the blood.


efmoroctocog alfa

mrdef: consists of factor VIII fused to the Fc domain of IgG1; for treatment of hemophilia A


talimogene laherparepvec

mrdef: Talimogene laherparepvec is a live, attenuated HSV-1 that has been genetically modified to express huGM-CSF. Talimogene laherparepvec has been genetically modified to replicate within tumors and to produce the immune stimulatory protein GM-CSF and causes lysis of tumors, followed by release of tumor-derived antigens, which together with virally derived GM-CSF may promote an antitumor immune response.


etelcalcetide

mrdef: Etelcalcetide is a synthetic peptide calcimimetic agent which reduces parathyroid hormone (PTH) secretion through binding and activation of the calcium-sensing receptor. The reduction in PTH is associated with a concomitant decrease in serum calcium and phosphate levels.


secondary hyperparathyroidism

description: Overproduction of parathyroid hormone in response to influence external to the parathyroid glands.


eteplirsen

mrdef: Eteplirsen is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein.


mevalonic aciduria

description: Mevalonic aciduria (MVA) is a rare, very severe form of mevalonate kinase deficiency (MKD) characterized by dysmorphic features, failure to thrive, psychomotor delay, ocular involvement, hypotonia, progressive ataxia, myopathy, and recurrent inflammatory episodes.


name: autosomal dominant familial periodic fever

description: A periodic fever syndrome, characterized by recurrent fever, arthralgia, myalgia and tender skin lesions lasting for 1 to 3 weeks, associated with skin, joint, ocular and serosal inflammation and complicated by secondary amyloidosis.


rubidium Rb-82 chloride

cd_formula: ClRb

mrdef: Rb-82 is analogous to potassium ion (K+) in its biochemical behavior and is rapidly extracted by the myocardium proportional to the blood flow. Rb+ participates in the sodium-potassium (Na+/K+) ion exchange pumps that are present in cell membranes. The intracellular uptake of Rb-82 requires maintenance of ionic gradient across cell membranes. Rb-82 radioactivity is increased in viable myocardium reflecting intracellular retention, while the tracer is cleared rapidly from necrotic or infarcted tissue.


gepirone

A member of the class of piperidones that is piperidine-2,6-dione substituted by a 4-[4-(pyrimidin-2-yl)piperazin-1-yl]butyl group at position 1 and two methyl groups at position 4.


apatinib

mrdef: Apatinib is an oral tyrosine kinase inhibitor approved in China for the treatment of patients with advanced metastatic gastric cancer.


deflazacort

mrdef: Deflazacort is a corticosteroid prodrug, whose active metabolite, 21-desDFZ, acts through the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects. The precise mechanism by which deflazacort exerts its therapeutic effects in patients with Duchenne muscular dystrophy is unknown.


tasonermin

mrdef: Tasonermin, is a copy of the human protein tumour necrosis factor alfa-1a (TNF-alfa). The exact way that TNF-alfa works against certain types of cancer is not fully understood, but it is thought that it can kill tumour cells directly, as well as destroy the blood vessels supplying tumours and stimulate the immune system to attack them. This leads to the tumour dying back and getting smaller, particularly when the medicine is combined with other cytotoxic (cell-killing) medicines and raised temperature.


brodalumab

mrdef: Brodalumab is a human monoclonal IgG2 antibody that selectively binds to human IL-17RA and inhibits its interactions with cytokines IL-17A, IL-17F, IL-17C, IL-17A/F heterodimer and IL-25. IL-17RA is a protein expressed on the cell surface and is a required component of receptor complexes utilized by multiple IL-17 family cytokines. Blocking IL17RA inhibits IL-17 cytokine-induced responses including the release of pro-inflammatory cytokines and chemokines.


sulesomab

mrdef: Sulesomab recognizes an antigenic structure shared by a surface glycoprotein (NCA-90) of granulocytes and the tumour marker, carcinoembryonic antigen (CEA). Sulesomab is indicated for diagnostic imaging for determining the location and extent of infection/inflammation in bone in patients with suspected osteomyelitis, including patients with diabetic foot ulcers.


telotristat ethyl

mrdef: Telotristat, the active metabolite of telotristat ethyl, is an inhibitor of tryptophan hydroxylase, which mediates the rate limiting step in serotonin biosynthesis. The in vitro inhibitory potency of telotristat towards tryptophan hydroxylase is 29 times higher than that of telotristat ethyl. Serotonin plays a role in mediating secretion, motility, inflammation, and sensation of the gastrointestinal tract, and is over-produced in patients with carcinoid syndrome. Through inhibition of tryptophan hydroxylase, telotristat and telotristat ethyl reduce the production of peripheral serotonin, and the frequency of carcinoid syndrome diarrhea.


crisantaspase

mrdef: Asparaginase Erwinia chrysanthemi catalyzes the deamidation of asparagine to aspartic acid and ammonia, resulting in a reduction in circulating levels of asparagine. The mechanism of action of ERWINAZE is thought to be based on the inability of leukemic cells to synthesize asparagine due to lack of asparagine synthetase activity, resulting in cytotoxicity specific for leukemic cells that depend on an exogenous source of amino acid asparagine for their protein metabolism and survival.


sivelestat

mrdef: inhibitor of neutrophil elastase; structure given in first source


acute lung injury

description: A condition of lung damage that is characterized by bilateral pulmonary infiltrates (pulmonary edema) rich in neutrophils, and in the absence of clinical heart failure. This can represent a spectrum of pulmonary lesions, endothelial and epithelial, due to numerous factors (physical, chemical, or biological).


limaprost

mrdef: Limaprost exerts potent effects on vasodilation, increase of blood flow and inhibition of platelet aggregation, and thereby has proven clinical effects on various ischemic symptoms such as ulcer, pain and feeling of coldness associated with thromboangiitis obliterans.


benperidol

mrdef: A butyrophenone with general properties similar to those of HALOPERIDOL. It has been used in the treatment of aberrant sexual behavior. (From Martindale, The Extra Pharmacopoeia, 30th ed, p567)


zuclopenthixol

mrdef: Zuclopenthixol is a potent neuroleptic of the thioxanthene series with a piperazine side-chain. The antipsychotic effect of neuroleptics is related to their dopamine receptor blocking effect. The thioxanthenes have a high affinity for both the adenylate cyclase coupled dopamine D1 receptors and for the dopamine D2 receptors; in the phenothiazine group the affinity for D1 receptors is much lower than that for D2 receptors, whereas butyrophenones, diphenylbutylpiperidines and benzamides only have affinity for D2 receptors.

description: The (Z)-isomer of clopenthixol.


flutoprazepam

mrdef: Flunitrazepam is a short-acting benzodiazepine with general properties similar to diazepam.


cytisine

mrdef: RN given refers to (R)-isomer; structure

description: An organic heterotricyclic compound that is the toxic principle in Laburnum seeds and is found in many members of the Fabaceae (legume, pea or bean) family. An acetylcholine agonist, it is widely used throughout Eastern Europe as an aid to giving up smoking.


ribociclib

mrdef: Ribociclib is an inhibitor of cyclin-dependent kinase (CDK) 4 and 6. These kinases are activated upon binding to D-cyclins and play a crucial role in signaling pathways which lead to cell cycle progression and cellular proliferation. The cyclin D-CDK4/6 complex regulates cell cycle progression through phosphorylation of the retinoblastoma protein (pRb). In vitro, ribociclib decreased pRb phosphorylation leading to arrest in the G1 phase of the cell cycle and reduced cell proliferation in breast cancer cell lines. In vivo, treatment with single agent ribociclib in a rat xenograft model with human tumor cells led to decreased tumor volumes, which correlated with inhibition of pRb phosphorylation. In studies using patient-derived estrogen receptor positive breast cancer xenograft models, combination of ribociclib and antiestrogen (e.g. letrozole) resulted in increased tumor growth inhibition compared to each drug alone.


trafermin

mrdef: used for treatment of stroke and coronary heart disease; RN amino acid sequence in first source


Atrophy of alveolar ridges

category: biolink:PhenotypicFeature biolink:DiseaseOrPhenotypicFeature biolink:BiologicalEntity biolink:ThingWithTaxon biolink:NamedThing


naldemedine

mrdef: Naldemedine is an opioid antagonist with binding affinities for mu-, delta-, and kappa-opioid receptors. Naldemedine functions as a peripherally-acting mu-opioid receptor antagonist in tissues such as the gastrointestinal tract, thereby decreasing the constipating effects of opioids.


avelumab

mrdef: Avelumab is a programmed death ligand-1 (PD-L1) blocking antibody. Avelumab is a human IgG1 lambda monoclonal antibody that has a molecular weight of approximately 147 kDa. PD-L1 may be expressed on tumor cells and tumor-infiltrating immune cells and can contribute to the inhibition of the anti-tumor immune response in the tumor microenvironment. Binding of PD-L1 to the PD-1 and B7.1 receptors found on T cells and antigen presenting cells suppresses cytotoxic T-cell activity, T-cell proliferation and cytokine production. Avelumab binds PD-L1 and blocks the interaction between PD-L1 and its receptors PD-1 and B7.1. This interaction releases the inhibitory effects of PD-L1 on the immune response resulting in the restoration of immune responses, including anti-tumor immune responses. Avelumab has also been shown to induce antibody-dependent cell-mediated cytotoxicity (ADCC) in vitro.


cutaneous neuroendocrine carcinoma

description: Cutaneous neuroendocrine carcinoma is a primary cutaneous cancer arising from a subset of skin neuroendocrine cells (Merkel cells, giving the name Merkel cell carcinoma (MCC)).


niraparib

mrdef: Niraparib is an inhibitor of poly(ADP-ribose) polymerase (PARP) enzymes, PARP-1 and PARP-2, which play a role in DNA repair. In vitro studies have shown that niraparib-induced cytotoxicity may involve inhibition of PARP enzymatic activity and increased formation of PARP-DNA complexes resulting in DNA damage, apoptosis and cell death. Increased niraparib-induced cytotoxicity was observed in tumor cell lines with or without deficiencies in BRCA1/2. Niraparib decreased tumor growth in mouse xenograft models of human cancer cell lines with deficiencies in BRCA1/2 and in human patient-derived xenograft tumor models with homologous recombination deficiency that had either mutated or wild type BRCA1/2.

description: A 2-[4-(piperidin-3-yl)phenyl]-2H-indazole-7-carboxamide that has S-configuration. It is a potent inhibitor of PARP1 and PARP2 (IC50 of 3.8 and 2.1 nM, respectively) and approved as a first-line maintenance treatment for women with advanced ovarian cancer after responding to platinum-based chemotherapy.


Carcinoma in situ of fallopian tube

description: Stage 0 includes: Tis, N0, M0.  Tis: Carcinoma in situ (limited to tubal mucosa). N0: No regional lymph node metastasis.  M0: No distant metastasis. FIGO no longer includes stage 0. (AJCC 7th ed.)


ocrelizumab

mrdef: The precise mechanism by which ocrelizumab exerts its therapeutic effects in multiple sclerosis is unknown, but is presumed to involve binding to CD20, a cell surface antigen present on pre-B and mature B lymphocytes. Following cell surface binding to B lymphocytes, ocrelizumab results in antibody-dependent cellular cytolysis and complement-mediated lysis.


primary progressive multiple sclerosis

description: A multiple sclerosis that is characterized by steady worsening of neurologic functioning, without any distinct relapses or periods of remission. The rate of progression may vary over time, with occasional plateaus or temporary improvements, but the progression is continuous.


dupilumab

mrdef: Dupilumab is a human monoclonal IgG4 antibody that inhibits interleukin-4 (IL-4) and interleukin-13 (IL-13) signaling by specifically binding to the IL-4Rα subunit shared by the IL-4 and IL-13 receptor complexes. Dupilumab inhibits IL-4 signaling via the Type I receptor and both IL-4 and IL-13 signaling through the Type II receptor. Blocking IL-4Rα with dupilumab inhibits IL-4 and IL-13 cytokine-induced responses, including the release of proinflammatory cytokines, chemokines and IgE.


sodium zirconium cyclosilicate

mrdef: Sodium zirconium cyclosilicate selectively binds potassium in exchange for hydrogen and sodium cations throughout the gastrointestinal (GI) tract and reduces the concentration of free potassium in the GI lumen. This lowers serum potassium levels by drawing potassium into the GI tract and increasing faecal potassium excretion to resolve hyperkalaemia.


valbenazine

mrdef: The mechanism of action of valbenazine in the treatment of tardive dyskinesia is unknown, but is thought to be mediated through the reversible inhibition of vesicular monoamine transporter 2 (VMAT2), a transporter that regulates monoamine uptake from the cytoplasm to the synaptic vesicle for storage and release.


simoctocog alfa

mrdef: Simoctocog alfa (human coagulation factor VIII (rDNA)) is a purified protein that has 1440 amino acids. The amino acid sequence is comparable to the 90 + 80 kDa form of human plasma factor VIII (i.e. B-domain deleted). Simoctocog alfa is produced by recombinant DNA technology in genetical human embryonic kidney (HEK) 293F cells.


eperisone

A racemate that is an equimolar mixture of (R)- and (S)-eperisone. It is used (as the hydrochloride salt) as a muscle relaxant for the symptomatic treatment of muscle spasm and spasticity.


Hyperkinetic movements

description: Motor hyperactivity with excessive movement of muscles of the body as a whole.


corifollitropin alfa

mrdef: Corifollitropin alfa is designed as a sustained follicle stimulant with the same pharmacodynamic profile as (rec)FSH, but with a markedly prolonged duration of FSH activity. Due to its ability to initiate and sustain multiple follicular growth for an entire week, a single subcutaneous injection of the recommended dose of Corifollitropin alfa may replace the first seven injections of any daily (rec)FSH preparation in a COS treatment cycle. The long duration of FSH activity was achieved by adding the carboxy-terminal peptide of the beta-subunit of human chorionic gonadotropin (hCG) to the beta-chain of human FSH. Corifollitropin alfa does not display any intrinsic LH/hCG activity.


guselkumab

mrdef: Guselkumab is a human monoclonal IgG1-lambda antibody that selectively binds to the p19 subunit of interleukin 23 (IL-23) and inhibits its interaction with the IL-23 receptor. IL-23 is a naturally occurring cytokine that is involved in normal inflammatory and immune responses. Guselkumab inhibits the release of proinflammatory cytokines and chemokines.


inotuzumab ozogamicin

mrdef: Inotuzumab ozogamicin is an antibody-drug conjugate (ADC) composed of a CD22-directed monoclonal antibody that is covalently linked to N-acetyl-gamma-calicheamicin dimethylhydrazide. Inotuzumab is a humanised immunoglobulin class G subtype 4 (IgG4) antibody that specifically recognises human CD22. The small molecule, N-acetyl-gamma-calicheamicin, is a cytotoxic product. N-acetyl-gamma-calicheamicin is covalently attached to the antibody via an acid-cleavable linker. Nonclinical data suggest that the anticancer activity of Inotuzumab ozogamicin is due to the binding of the ADC to CD22-expressing tumour cells, followed by internalisation of the ADC-CD22 complex, and the intracellular release of N-acetyl-gamma-calicheamicin dimethylhydrazide via hydrolytic cleavage of the linker. Activation of N-acetyl-gamma-calicheamicin dimethylhydrazide induces double-stranded DNA breaks, subsequently inducing cell cycle arrest and apoptotic cell death.


pemafibrate

mrdef: Pemafibrate is a selective PPAR-alpha modulator (SPPARM-alpha) that has antihyperlipidaemic activity.

description: A member of the class of 1,3-benzoxazoles that is 1,3-benzoxazol-2-amine in which the amino hydrogens are replaced by 3-[(1R)-1-carboxypropoxy]benzyl and 3-(4-methoxyphenoxy)propyl groups. It is a selective peroxisome proliferator-activated receptor (PPAR)-alpha agonist that is used for the treatment of hyperlipidaemia.


acute cystitis

description: An acute infection of the bladder. It is usually caused by bacteria. Signs and symptoms include increased frequency of urination, pain or burning during urination, fever, cloudy or bloody urine, and suprapubic pain.


zabofloxacin

mrdef: Zabofloxacin is a fluoroquinolone antibiotic with enhanced in-vitro activity against Streptococcus pneumoniae, including strains resistant to other antibiotics. The spectrum of activity of Zabofloxacin includes those bacterial strains that are responsible for most community-acquired respiratory infections.


name: Acute infective exacerbation of chronic obstructive pulmonary disease


imrecoxib

mrdef: Imrecoxib is a selective cyclooxygenase-2 (COX-2) inhibitor indicated for treatment of osteoarthritis.


enasidenib

mrdef: Enasidenib is a small molecule inhibitor of the isocitrate dehydrogenase 2 (IDH2) enzyme. Enasidenib targets the mutant IDH2 variants R140Q, R172S, and R172K at approximately 40-fold lower concentrations than the wild-type enzyme in vitro. Inhibition of the mutant IDH2 enzyme by enasidenib led to decreased 2-hydroxyglutarate (2-HG) levels and induced myeloid differentiation in vitro and in vivo in mouse xenograft models of IDH2 mutated AML. In blood samples from patients with AML with mutated IDH2, enasidenib decreased 2-HG levels, reduced blast counts and increased percentages of mature myeloid cells.

description: A 1,3,5-triazine which is substituted by (2-hydroxy-2-methylpropyl)nitrilo, 6-(trifluoromethyl)pyridin-2-yl and [2-(trifluoromethyl)pyridin-4-yl]nitrilo groups at positions 2,4 and 6, respectively. It is an isocitrate dehydrogenase-2 (IDH2) inhibitor which has been approved for the treatment of adults with relapsed or refractory acute myeloid leukaemia (AML).


neratinib

mrdef: Neratinib is a kinase inhibitor that irreversibly binds to Epidermal Growth Factor Receptor (EGFR), Human Epidermal Growth Factor Receptor 2 (HER2), and HER4. In vitro, neratinib reduces EGFR and HER2 autophosphorylation, downstream MAPK and AKT signaling pathways, and showed antitumor activity in EGFR and/or HER2 expressing carcinoma cell lines. Neratinib human metabolites M3, M6, M7 and M11 inhibited the activity of EGFR, HER2 and HER4 in vitro. In vivo, oral administration of neratinib inhibited tumor growth in mouse xenograft models with tumor cell lines expressing HER2 and EGFR.

description: A quinoline compound having a cyano group at the 3-position, a 3-chloro-4-(2-pyridylmethoxy)anilino group at the 4-position, a 4-dimethylamino-trans-but-2-enoylamido group at the 6-position, and an ethoxy group at the 7-position.


chronic graft versus host disease

description: Chronic graft versus host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted donor cells attack the transplant recipient's body. Symptoms may include skin rash, mouth sores, dry eyes, liver inflammation, development of scar tissue in the skin and joints, and damage to the lungs. The exact cause of chronic GVHD is unknown.It likely results from a complex immune-mediated interaction between the donor and recipient cells.Chronic GVHD is treated with prednisone or other similar anti-inflammatory or immunosuppressive medications.


macimorelin

mrdef: Macimorelin stimulates GH release by activating growth hormone secretagogue receptors present in the pituitary and hypothalamus.


angiotensin II

mrdef: Angiotensin II is a naturally occurring peptide hormone of the renin-angiotensin-aldosterone system (RAAS) that causes vasoconstriction and an increase in blood pressure. Angiotensin II raises blood pressure by vasoconstriction and increased aldosterone release. Direct action of angiotensin II on the vessel wall is mediated by binding to the G-protein-coupled angiotensin II receptor type 1 on vascular smooth muscle cells, which stimulates Ca2+/calmodulin-dependent phosphorylation of myosin and causes smooth muscle contraction.

description: An angiotensin II that acts on the central nervous system (PDB entry: 1N9V).


ertugliflozin

mrdef: SGLT2 is the predominant transporter responsible for reabsorption of glucose from the glomerular filtrate back into the circulation. Ertugliflozin is an inhibitor of SGLT2. By inhibiting SGLT2, ertugliflozin reduces renal reabsorption of filtered glucose and lowers the renal threshold for glucose, and thereby increases urinary glucose excretion.


padeliporfin

mrdef: Padeliporfin is retained within the vascular system. When activated with 753 nm wavelength laser light, padeliporfin triggers a cascade of pathophysiological events resulting in focal necrosis within a few days. Activation within the illuminated tumour vasculature, generates oxygen radicals causing local hypoxia that induces the release of nitric oxide radicals. This results in transient arterial vasodilatation that triggers the release of the vasoconstrictor, endothelin-1. Rapid consumption of the NO radicals, by oxygen radicals, leads to the formation of reactive nitrogen species (RNS) (e.g. peroxynitrite), in parallel to arterial constriction. In addition, impaired deformability is thought to enhance erythrocyte aggregability and formation of blood clots at the interface of the arterial supply (feeding arteries) and tumour microcirculation, results in occlusion of the tumour vasculature. This is enhanced by RNS-induced endothelial cell apoptosis and initiation of self-propagated tumour cells necrosis through peroxidation of their membrane.


prostate adenocarcinoma

description: A carcinoma that arises from glandular epithelial cells of the prostate gland


tezacaftor

mrdef: Tezacaftor facilitates the cellular processing and trafficking of normal and select mutant forms of CFTR (including F508del-CFTR) to increase the amount of mature CFTR protein delivered to the cell surface.


bictegravir

mrdef: Bictegravir inhibits the strand transfer activity of HIV-1 integrase (integrase strand transfer inhibitor; INSTI), an HIV-1 encoded enzyme that is required for viral replication. Inhibition of integrase prevents the integration of linear HIV-1 DNA into host genomic DNA, blocking the formation of the HIV-1 provirus and propagation of the virus.

description: A monocarboxylic acid amide obtained by formal condensation of the carboxy group of (2R,5S,13aR)-8-hydroxy-7,9-dioxo-2,3,4,5,7,9,13,13a-octahydro-2,5-methanopyrido[1',2':4,5]pyrazino[2,1-b][1,3]oxazepine-10-carboxylic acid with the amino group of 2,4,6-trifluorobenzylamine. It is a second-generation integrase strand transfer inhibitor (INSTI) and used (as its sodium salt) for the treatment of HIV-1.


lutetium (177Lu) oxodotreotide

mrdef: Lutetium Lu 177 dotatate binds to somatostatin receptors with highest affinity for subtype 2 receptors (SSTR2). Upon binding to somatostatin receptor expressing cells, including malignant somatostatin receptor-positive tumors, the compound is internalized. The beta emission from Lu 177 induces cellular damage by formation of free radicals in somatostatin receptor-positive cells and in neighboring cells.


neuroendocrine neoplasm

description: Endocrine tumors, also referred to as neuroendocrine tumors (NETs), are defined by a common phenotype which is characterized by the expression of general markers (neuron specific enolase, chromogranin, synaptophysin) and hormone secretion products. These tumors may be localized in any part of the body and are generally discovered in non-specific situations, i.e. not immediately suggestive of NETs (tests for inherited predisposition to tumors or for a clinical syndrome caused by abnormal hormone secretion).


elobixibat

mrdef: Elobixibat is an IBAT inhibitor approved in Japan for the treatment of chronic constipation. IBAT is the bile acid/sodium symporter responsible for the reuptake of bile acids in the ileum which is the initial step in the enterohepatic circulation. By inhibiting the uptake of bile acids, elobixibat increases the bile acid concentration in the gut, and this accelerates intestinal passage and softens the stool.


Chronic constipation

description: Constipation for longer than three months with fewer than 3 bowel movements per week, straining, lumpy or hard stools, and a sensation of anorectal obstruction or incomplete defecation.


apalutamide

mrdef: Apalutamide is an Androgen Receptor (AR) inhibitor that binds directly to the ligand-binding domain of the AR. Apalutamide inhibits AR nuclear translocation, inhibits DNA binding, and impedes AR-mediated transcription. A major metabolite, N-desmethyl apalutamide, is a less potent inhibitor of AR, and exhibited one-third the activity of apalutamide in an in vitro transcriptional reporter assay. Apalutamide administration caused decreased tumor cell proliferation and increased apoptosis leading to decreased tumor volume in mouse xenograft models of prostate cancer.


tecovirimat

mrdef: Tecovirimat targets and inhibits the activity of the orthopoxvirus VP37 protein (encoded by and highly conserved in all members of the orthopoxvirus genus) and blocks its interaction with cellular Rab9 GTPase and TIP47, which prevents the formation of egress-competent enveloped virions necessary for cell-to-cell and long-range dissemination of virus.


smallpox

description: A condition that is caused by infection with Variola, and that is characterized by small, raised bumps.


ivosidenib

mrdef: Ivosidenib is a small molecule inhibitor that targets the mutant isocitrate dehydrogenase 1 (IDH1) enzyme. Susceptible IDH1 mutations are defined as those leading to increased levels of 2-hydroxyglutarate(2-HG) in the leukemia cells and where efficacy is predicted by 1) clinically meaningful remissions with the recommended dose of ivosidenib and/or 2) inhibition of mutant IDH1 enzymatic activity at concentrations of ivosidenib sustainable at the recommended dosage according to validated methods. The most common of such mutations are R132H and R132C substitutions.

description: A tertiary carboxamide resulting from the formal condensation of the carboxy group of (2S)-1-(4-cyanopyridin-2-yl)-5-oxopyrrolidine-2-carboxylic acid with the secondary amino group of (2S)-2-(2-chlorophenyl)-N-(3,3-difluorocyclobutyl)-2-[(5-fluoropyridin-3-yl)amino]acetamide. It is approved by the FDA for the treatment of acute myeloid leukemia (AML) in patients with an isocitrate dehydrogenase-1 (IDH1) mutation.


elagolix

mrdef: Elagolix is a GnRH receptor antagonist that inhibits endogenous GnRH signaling by binding competitively to GnRH receptors in the pituitary gland. Administration of ORILISSA results in dose-dependent suppression of luteinizing hormone (LH) and follicle-stimulating hormone (FSH), leading to decreased blood concentrations of the ovarian sex hormones, estradiol and progesterone.


eravacycline dihydrochloride

mrdef: Eravacycline is a fluorocycline antibacterial within the tetracycline class of antibacterial drugs. Eravacycline disrupts bacterial protein synthesis by binding to the 30S ribosomal subunit thus preventing the incorporation of amino acid residues into elongating peptide chains.


doravirine

mrdef: Doravirine is a pyridinone non-nucleoside reverse transcriptase inhibitor of HIV-1 and inhibits HIV-1 replication by non-competitive inhibition of HIV-1 reverse transcriptase (RT). Doravirine does not inhibit the human cellular DNA polymerases alpha, beta, and mitochondrial DNA polymerase gamma.


duvelisib

mrdef: Duvelisib is an inhibitor of PI3K with inhibitory activity predominantly against PI3K-delta and PI3K-gamma isoforms expressed in normal and malignant B-cells. Duvelisib induced growth inhibition and reduced viability in cell lines derived from malignant B-cells and in primary CLL tumor cells. Duvelisib inhibits several key cell-signaling pathways, including B-cell receptor signaling and CXCR12-mediated chemotaxis of malignant B-cells. Additionally, duvelisib inhibits CXCL12-induced T cell migration and M-CSF and IL-4 driven M2 polarization of macrophages.


sarecycline

mrdef: The mechanism of action of sarecycline in treating acne vulgaris is not known.


omadacycline

mrdef: Omadacycline is an aminomethylcycline antibacterial within the tetracycline class of antibacterial drugs.  Omadacycline binds to the 30S ribosomal subunit and blocks protein synthesis.  Omadacycline is active in vitro against Gram positive bacteria expressing tetracycline resistance active efflux pumps (tetK and tet L) and ribosomal protection proteins (tet M).  In general, omadacycline is considered bacteriostatic; however, omadacycline has demonstrated bactericidal activity against some isolates of S. pneumoniae and H. influenzae.


talazoparib tosylate

mrdef: Talazoparib is an inhibitor of poly (ADP-ribose) polymerase (PARP) enzymes, including PARP1 and PARP2, which play a role in DNA repair. In vitro studies with cancer cell lines that harbored defects in DNA repair genes, including BRCA 1 and 2, have shown that talazoparib-induced cytotoxicity may involve inhibition of PARP enzymatic activity and increased formation of PARP-DNA complexes resulting in DNA damage, decreased cell proliferation, and apoptosis. Talazoparib anti-tumor activity was observed in human patient-derived xenograft breast cancer tumor models that expressed mutated or wild-type BRCA 1 and 2.

description: Talazoparib is an orally available small molecule inhibitor of the DNA repair enzyme poly ADP-ribose polymerase (PARP) which is used as an antineoplastic agent in the treatment of selected cases of breast cancer. Talazoparib is associated with a moderate rate of serum aminotransferase elevations during therapy and is suspected to cause rare instances of clinically apparent acute liver injury.


name: Germline BRCA-mutated, HER2-negative metastatic breast cancer


dacomitinib

mrdef: Dacomitinib is an irreversible inhibitor of the kinase activity of the human EGFR family (EGFR/HER1, HER2, and HER4) and certain EGFR activating mutations (exon 19 deletion or the exon 21 L858R substitution mutation). In vitro dacomitinib also inhibited the activity of DDR1, EPHA6, LCK, DDR2, and MNK1 at clinically relevant concentrations.

description: A member of the class of quinazolines that is 7-methoxyquinazoline-4,6-diamine in which the amino group at position 4 is substituted by a 3-chloro-4-fluorophenyl group and the amino group at position 6 is substituted by an (E)-4-(piperidin-1-yl)but-2-enoyl group.


lorlatinib

mrdef: Lorlatinib is a kinase inhibitor with in vitro activity against ALK and ROS1 as well as TYK1, FER, FPS, TRKA, TRKB, TRKC, FAK, FAK2, and ACK. Lorlatinib demonstrated in vitro activity against multiple mutant forms of the ALK enzyme, including some mutations detected in tumors at the time of disease progression on crizotinib and other ALK inhibitors.

description: A cyclic ether that is 16,17-dihydro-2H-8,4-(metheno)pyrazolo[4,3-h][2,5,11]benzoxadiazacyclotetradecin-15(10H)-one substituted by methyl groups at positions 2 and 10R, and by cyano, amino and fluoro groups at positions 3, 7 and 12 respectively. It is a small molecule inhibitor of ALK and ROS1 kinase developed by Pfizer for the treatment of ALK-positive non-small cell lung cancer.


revefenacin

mrdef: Revefenacin is a long-acting muscarinic antagonist, which is often referred to as an anticholinergic. It has similar affinity to the subtypes of muscarinic receptors M1 to M5. In the airways, it exhibits pharmacological effects through inhibition of M3 receptor at the smooth muscle leading to bronchodilation. The competitive and reversible nature of antagonism was shown with human and animal origin receptors and isolated organ preparations. In preclinical in vitro as well as in vivo models, prevention of methacholine- and acetylcholine-induced bronchoconstrictive effects was dose-dependent and lasted longer than 24 hours. The clinical relevance of these findings is unknown. The bronchodilation following inhalation of revefenacin is predominantly a site-specific effect.


glasdegib

mrdef: Glasdegib is an inhibitor of the Hedgehog pathway. Glasdegib binds to and inhibits Smoothened, a transmembrane protein involved in hedgehog signal transduction.

description: A member of the class of benzimidazoles that is 1H-benzimidazole which is substituted by a (2R,4S)-4-{[(4-cyanophenyl)carbamoyl]amino}-1-methylpiperidin-2-yl group at position 2. It is a hedgehog signalling pathway inhibitor that acts by binding to Smoothened (SMO) receptors and blocking signal transduction (IC50 = 5 nM). It is used in combination with low-dose cytarabine, for the treatment of newly-diagnosed acute myeloid leukemia (AML) in adult patients (aged >= 75 years), or who have medical conditions that prevent the use of standard chemotherapy.


gilteritinib

mrdef: Gilteritinib is a small molecule that inhibits multiple receptor tyrosine kinases, including FMS-like tyrosine kinase 3 (FLT3). Gilteritinib demonstrated the ability to inhibit FLT3 receptor signaling and proliferation in cells exogenously expressing FLT3 including FLT3-ITD, tyrosine kinase domain mutations (TKD) FLT3-D835Y and FLT3-ITD-D835Y, and it induced apoptosis in leukemic cells expressing FLT3-ITD.

description: A member of the class of pyrazines that is pyrazine-2-carboxamide which is substituted by {3-methoxy-4-[4-(4-methylpiperazin-1-yl)piperidin-1-yl]phenyl}nitrilo, (oxan-4-yl)nitrilo and ethyl groups at positions 3,5 and 6, respectively. It is a potent inhibitor of FLT3 and AXL tyrosine kinase receptors (IC50 = 0.29 nM and 0.73 nM, respectively). Approved by the FDA for the treatment of acute myeloid leukemia in patients who have a FLT3 gene mutation.


evocalcet

mrdef: Evocalcet is a new type of oral calcimimetics agent. The drug suppresses parathyroid hormone secretion by acting on the calcium receptors on parathyroid gland cells.


rifamycin SV

description: A member of the class of rifamycins that exhibits antibiotic and antitubercular properties.


triclabendazole

mrdef: Benzimidazole antiplatyhelmintic agent that is used for the treatment of FASCIOLIASIS and PARAGONIMIASIS.


fascioliasis

description: A parasitic infection that is caused by liver flukes, usually Fasciola hepatica, of sheep, goats, and cattle. Humans become infected by eating uncooked, infested aquatic vegetation (classically watercress). adult flukes inhabit the bile ducts, gallbladder, and occasionally ectopic sites. Symptoms arise secondary to inflammatory response or obstruction.


ATTRV122I amyloidosis

description: A rare hereditary Transthyretin (TTR)-related systemic amyloidosis (ATTR) with predominant cardiac involvement resulting from myocardial infiltration of abnormal amyloid protein.


brexanolone

mrdef: Brexanolone is a neuroactive steroid gamma-aminobutyric acid (GABA) A receptor positive modulator indicated for the treatment of postpartum depression (PPD) in adults.


solriamfetol

mrdef: The mechanism of action of solriamfetol to improve wakefulness in patients with excessive daytime sleepiness associated with narcolepsy or obstructive sleep apnea is unclear. However, solriamfetol binds to the dopamine transporter and norepinephrine transporter with low affinity and inhibits the reuptake of dopamine and norepinephrine with low potency. Solriamfetol has no appreciable binding affinity for the serotonin transporter (Ki=81.5 μM) and does not inhibit serotonin reuptake (IC50 > 100 μM). Solriamfetol improves wakefulness in patients with excessive daytime sleepiness associated with narcolepsy or obstructive sleep apnea is unclear. However, its efficacy could be mediated through its activity as a dopamine and norepinephrine reuptake inhibitor (DNRI).


name: Excessive daytime sleepiness - normal night sleep


obstructive sleep apnea syndrome

description: Cessation of air flow during sleep due to upper airway obstruction.


siponimod

mrdef: Siponimod is a sphingosine-1-phosphate (S1P) receptor modulator. Siponimod binds with high affinity to S1P receptors 1 and 5. Siponimod blocks the capacity of lymphocytes to egress from lymph nodes, reducing the number of lymphocytes in peripheral blood. The mechanism by which siponimod exerts therapeutic effects  in multiple sclerosis is unknown, but may involve reduction of lymphocyte migration into the central nervous system.


alpelisib

mrdef: Alpelisib is an inhibitor of phosphatidylinositol-3- kinase (PI3K) with inhibitory activity predominantly against PI3Kalpha. Gain-of-function mutations in the gene encoding the catalytic alpha-subunit of PI3K (PIK3CA) lead to activation of PI3Kalpha and Akt-signaling, cellular transformation and the generation of tumors in in vitro and in vivo models.


bremelanotide

mrdef: Bremelanotide is a melanocortin receptor (MCR) agonist that nonselectively activates several receptor subtypes with the following order of potency: MC1R, MC4R, MC3R, MC5R, MC2R. The mechanism by which VYLEESI improves HSDD in women is unknown. The MC1R is expressed on melanocytes; binding at this receptor leads to melanin expression and increased pigmentation


prabotulinumtoxinA

mrdef: PrabotulinumtoxinA-xvfs is an acetylcholine release inhibitor and a neuromuscular blocking agent


onasemnogene abeparvovec

mrdef: ZOLGENSMA is a recombinant AAV9-based gene therapy designed to deliver a copy of the gene encoding the human SMN protein. SMA is caused by a bi-allelic mutation in the SMN1 gene, which results in insufficient SMN protein expression. Intravenous administration of ZOLGENSMA that results in cell transduction and expression of the SMN protein has been observed in two human case studies


caplacizumab

mrdef: In patients with aTTP, levels of a substance called von Willebrand factor are increased. von Willebrand factor acts on platelets to cause them to stick together and form blood clots. Caplacizumab, the active substance in Cablivi, is a nanobody (a small antibody) which has been designed to attach to von Willebrand factor in a way that stops it acting on platelets. This reduces platelets sticking together and forming clots in blood vessels and, as a result, platelet levels in the blood rise because they are no longer taken up to form clots.


lanadelumab

mrdef: Lanadelumab-flyo is a fully human monoclonal antibody (IgG1/kappa-light chain) that binds plasma kallikrein and inhibits its proteolytic activity. Plasma kallikrein is a protease that cleaves high-molecular-weight-kininogen (HMWK) to generate cleaved HMWK (cHMWK) and bradykinin, a potent vasodilator that increases vascular permeability resulting in swelling and pain associated with HAE.In patients with HAE due to C1-inhibitor (C1-INH) deficiency or dysfunction, normal regulation of plasma kallikrein activity is not present, which leads to uncontrolled increases in plasma kallikrein activity and results in angioedema attacks. Lanadelumab-flyo decreases plasma kallikrein activity to control excess bradykinin generation in patients with HAE.


condoliase

mrdef: Condoliase degrades chondroitin sulfate, chondroitin, and hyaluronic acid and also improves the clinical manifestations of herniation by lowering the internal pressure of the intervertebral disc due to dissolving the glycosaminoglycans within the nucleus pulposus of the intervertebral disc, and lowering the water-holding capacity of the nucleus pulposus.


vibegron

mrdef: Vibegron is selectively binds to the beta 3 adrenergic receptor (beta3 AR) in bladder smooth muscle, resulting in relaxation of the detrusor muscle.

description: A pyrrolopyrimidine obtained by formal condensation of the carboxy group of (6S)-4-oxo-4,6,7,8-tetrahydropyrrolo[1,2-a]pyrimidine-6-carboxylic acid with the amino group of (R)-[(2R,5S)-5-(4-aminobenzyl)pyrrolidin-2-yl](phenyl)methanol. It is a beta3-adrenergic receptor agonist currently in clinical development for the treatment of patients with overactive bladder.


omidenepag isopropyl

mrdef: Omidenepag isopropyl is a prodrug that is converted by hydrolysis to omidenepag, the active form that it is thought to lower ophthalmic pressure via a dual mechanism of action, increasing both the trabecular outfow facility and the uveoscleral outfow as a result of EP2 receptor stimulation, thereby promoting aqueous outfow.


patisiran

mrdef: Patisiran is a double-stranded siRNA that causes degradation of mutant and wild-type TTR mRNA through RNA interference, which results in a reduction of serum TTR protein and TTR protein deposits in tissues.


Amyloid Neuropathies

description: Neuropathy caused by amyloid deposition in the peripheral nerves.


moxetumomab pasudotox

mrdef: Moxetumomab pasudotox-tdfk is a CD22-directed cytotoxin. Moxetumomab pasudotox-tdfk binds CD22 on the cell surface of B-cells and is internalized. Moxetumomab pasudotox -tdfk internalization results in ADP-ribosylation of elongation factor 2, inhibition of protein synthesis, and apoptotic cell death.


fremanezumab

mrdef: Fremanezumab-vfrm is a humanized monoclonal antibody that binds to calcitonin gene-related peptide (CGRP) ligand and blocks its binding to the receptor.


galcanezumab

mrdef: Galcanezumab-gnlm  is a humanized  monoclonal  antibody that  binds to calcitonin gene-related  peptide (CGRP)  ligand and blocks its binding to the receptor.


cemiplimab

mrdef: Cemiplimab-rwlc is a recombinant human immunoglobulin G4 (IgG4) monoclonal antibody that binds to PD-1 and blocks its interaction with PD-L1 and PD-L2, releasing PD-1 pathway mediated inhibition of the immune response, including the anti-tumor immune response. In syngeneic mouse tumor models, blocking PD-1 activity resulted in decreased tumor growth.


elapegademase

mrdef: Elapegademase-lvlr provides an exogenous source of ADA enzyme that is associated with a decrease in toxic adenosine and deoxyadenosine nucleotides levels as well as an increase in lymphocyte number.


name: SCID (severe combined immunodeficiency) due to absent adenosine deaminase


inotersen

mrdef: Inotersen is an antisense oligonucleotide that causes degradation of mutant and wild-type TTR mRNA through binding to the TTR mRNA, which results in a reduction of serum TTR protein and TTR protein deposits in tissues.


description: An instance of hemophagocytic lymphohistiocytosis that is caused by an inherited genomic modification in an individual.


ravulizumab

mrdef: Ravulizumab-cwvz is a terminal complement inhibitor that specifically binds to the complement protein C5 with high affinity, thereby inhibiting its cleavage to C5a (the proinflammatory anaphylatoxin) and C5b (the initiating subunit of the terminal complement complex [C5b-9]) and preventing the generation of the terminal complement complex C5b9. ULTOMIRIS inhibits terminal complement-mediated intravascular hemolysis in patients with PNH.


meningococcal infection

description: Infections with bacteria of the species neisseria meningitidis.


tagraxofusp

mrdef: Tagraxofusp-erzs is a CD123-directed cytotoxin composed of recombinant human interleukin-3 (IL-3) and truncated diphtheria toxin (DT) fusion protein that inhibits protein synthesis and causes cell death in CD123-expressing cells.


CD4+/CD56+ hematodermic neoplasm

description: An aggressive immature hematologic neoplasm formerly known as blastic NK cell lymphoma, composed of cells with a lymphoblast-like morphology. Recent evidence suggests derivation from a plasmacytoid monocyte. Patients present with cutaneous tumors and bone marrow involvement.


larotrectinib

mrdef: Larotrectinib is an inhibitor of the tropomyosin receptor kinases (TRK), TRKA, TRKB, and TRKC. In a broad panel of purified enzyme assays, larotrectinib inhibited TRKA, TRKB, and TRKC with IC50 values between 5-11 nM. One other kinase TNK2 was inhibited at approximately 100-fold higher concentration. TRKA, B, and C are encoded by the genes NTRK1, NTRK2, and NTRK3.Chromosomal rearrangements involving in-frame fusions of these genes with various partners can result in constitutively-activated chimeric TRK fusion proteins that can act as an oncogenic driver, promoting cell proliferation and survival in tumor cell lines.


polatuzumab vedotin

mrdef: Polatuzumab vedotin-piiq is a CD79b-directed antibody-drug conjugate with activity against dividing B cells. The small molecule, MMAE is an anti-mitotic agent covalently attached to the antibody via a cleavable linker. The monoclonal antibody binds to CD79b, a B-cell specific surface protein, which is a component of the B-cell receptor. Upon binding CD79b, polatuzumab vedotin-piiq is internalized, and the linker is cleaved by lysosomal proteases to enable intracellular delivery of MMAE. MMAE binds to microtubules and kills dividing cells by inhibiting cell division and inducing apoptosis


darolutamide

mrdef: Darolutamide is an androgen receptor (AR) inhibitor. Darolutamide competitively inhibits androgen binding, AR nuclear translocation, and AR-mediated transcription. A major metabolite, keto-darolutamide, exhibited similar in vitro activity to darolutamide. In addition, darolutamide functioned as a progesterone receptor (PR) antagonist in vitro (approximately 1% activity compared to AR). Darolutamide decreased prostate cancer cell proliferation in vitro and tumor volume in mouse xenograft models of prostate cancer


castration-resistant prostate carcinoma

description: A prostate carcinoma that is characterized by continued growth and spread despite the surgical removal of the testes or medical intervention to block androgen production.


volanesorsen

mrdef: Volanesorsen is an antisense oligonucleotide designed to inhibit the formation of apoC-III, a protein that is recognised to regulate both triglyceride metabolism and hepatic clearance of chylomicrons and other triglyceride-rich lipoproteins. The selective binding of volanesorsen to the apoC-III messenger ribonucleic acid (mRNA) within the 3′ untranslated region at base position 489-508 causes the degradation of the mRNA. This binding prevents translation of the protein apoC-III, thus removing an inhibitor of triglyceride clearance and enabling metabolism through an LPL-independent pathway


andexanet alfa

mrdef: Andexanet alfa is a recombinant form of human FXa protein that has been modified to lack FXa enzymatic activity. The active site serine was substituted with alanine, rendering the molecule unable to cleave and activate prothrombin, and the gamma-carboxyglutamic acid (Gla) domain was removed to eliminate the ability of the protein to assemble into the prothrombinase complex, thus removing any anti-coagulant effects. Andexanet alfa is a specific reversal agent for FXa inhibitors. The predominant mechanism of action is the binding and sequestration of the FXa inhibitor, although there may be a minor contribution from the inhibition of tissue factor pathway inhibitor (TFPI) activity through binding to TFPI. The interaction between andexanet alfa and TFPI has not been fully characterized. Andexanet alfa binds direct FXa inhibitors with high affinity, making them unavailable to exert their anticoagulant effects


name: Direct acting anticoagulant adverse reaction


risankizumab

mrdef: Skyrizi is a medicine used to treat plaque psoriasis, a disease causing red, scaly patches on the skin. Indicated for the treatment of moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy


ropeginterferon alfa-2b

mrdef: Ropeginterferon alfa-2b is a recombinant interferon alfa-2b conjugated with a two-arm methoxypolyethylene glycol (mPEG). It belongs to the class of type I interferons which exhibit their cellular effects by binding to a transmembrane receptor termed interferon alfa receptor (IFNAR). Binding to IFNAR initiates a downstream signalling cascade through the activation of kinases, in particular Janus kinase 1 (JAK1) and tyrosine kinase 2 (TYK2) and activator of transcription (STAT) proteins. Nuclear translocation of STAT proteins controls distinct gene-expression programs and exhibit various cellular effects. Interferon alfa was shown to have an inhibitory effect on the proliferation of hematopoietic and bone marrow fibroblast progenitor cells and antagonised the action of growth factors and other cytokines that have a role in the development of myelofibrosis. These actions may be involved in the therapeutic effects of interferon alfa in polycythaemia vera.Further, it was demonstrated that interferon alfa is able to decrease the mutated JAK2V617F allele burden in patients with polycythaemia vera (a V617F point mutation in the JAK2 kinase is a hallmark of polycythaemia vera and is  present in approximately 95% of patients)


sotagliflozin

mrdef: Sotagliflozin is a dual inhibitor of sodium glucose cotransporter type 1 (SGLT1) and SGLT2. Local intestinal inhibition of SGLT1, the major transporter for glucose absorption, delays and reduces glucose absorption in the proximal intestine, resulting in a blunting and delay of postprandial hyperglycaemia. SGLT2 is the predominant transporter responsible for reabsorption of glucose from the glomerular filtrate back into the circulation. By inhibiting SGLT2, sotagliflozin reduces renal reabsorption of filtered glucose and lowers the renal threshold for glucose, and thereby increases urinary glucose excretion

description: An S-glycosyl compound that is 1-thio-beta-L-xylopyranose in which the anomeric hydroxy group is replaced by a 4-chloro-3-[(4-ethoxyphenyl)methyl]phenyl group and the thiol is replaced by a methylsulfanediyl group. It is an inhibitor of SGLT1 and SGLT2 that is approved to reduce the risk of cardiovascular death, hospitalization for heart failure, and urgent heart failure visit in adults with heart failure or type 2 diabetes mellitus, chronic kidney disease, and other cardiovascular risk.


pegvaliase

mrdef: The active substance in Palynziq, pegvaliase, is a bacterial enzyme that can break down phenylalanine, thereby stopping phenylalanine from building up in the body and helping to relieve the symptoms of phenylketonuria. The enzyme in pegvaliase is ‘pegylated’ (attached to a chemical called PEG), allowing it to remain in the body and to act for longer


quizartinib

mrdef: Quizartinib is the most selective type II FLT3 inhibitor and has shown the strongest single-agent activity in patient population with R/R-AML (acute myeloid leukemia) with FLT3 mutations. The FLT3 tyrosine kinase inhibitors act as direct inhibitors of FLT3 via competitive inhibition of ATP-binding sites in the FLT3 receptor. Type II FLT3 inhibitors bind the FLT3 receptor in the inactive conformation in a region adjacent to the ATP-binding domain. As a result of this binding affinity, these inhibitors prevent activity of ITD (the internal tandem duplication) mutations but do not target TKD (the tyrosine kinase domain) mutations

description: A member of the class of phenylureas that is urea in which one of the amino groups has been substituted by a 5-tert-butyl-1,2-oxazol-3-yl group while the other has been substituted by a phenyl group substituted at the para- position by an imidazo[2,1-b][1,3]benzothiazol-2-yl group that, in turn, is substituted at position 7 by a 2-(morpholin-4-yl)ethoxy group.


name: Acute myeloid leukemia with FMS-like tyrosine kinase-3 mutation


name: Monoamine oxidase inhibitor (disposition)


fallopian tube neoplasm

description: A benign or malignant neoplasm affecting the fallopian tube. Representative examples of benign neoplasms include papilloma, adenofibroma, and leiomyoma. Representative examples of malignant neoplasms include carcinoma, carcinosarcoma, and adenosarcoma.


esketamine

mrdef: Esketamine is the S-enantiomer of racemic ketamine. It is a non-selective, non-competitive, antagonist of the N-methyl-D-aspartate (NMDA) receptor, an ionotropic glutamate receptor. Through NMDA receptor antagonism, esketamine produces a transient increase in glutamate release leading to increases in alpha-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid receptor (AMPAR) stimulation and subsequently to increases in neurotrophic signalling which may contribute to the restoration of synaptic function in these brain regions involved with the regulation of mood and emotional behaviour. Restoration of dopaminergic neurotransmission in brain regions involved in the reward and motivation, and decreased stimulation of brain regions involved in anhedonia, may contribute to the rapid response.

description: The S- (more active) enantiomer of ketamine.


erdafitinib

mrdef: Erdafitinib inhibited FGFR phosphorylation and signaling and decreased cell viability in cell lines expressing FGFR genetic alterations, including point mutations, amplifications, and fusions. Erdafitinib demonstrated antitumor activity in FGFR-expressing cell lines and xenograft models derived from tumor types, including bladder cancer.


Metastatic urothelial carcinoma

description: A carcinoma that arises from the urothelium and has spread from its original site of growth to another anatomic site.


selinexor

mrdef: Selinexor reversibly inhibits nuclear export of tumor suppressor proteins (TSPs), growth regulators, and mRNAs of oncogenic proteins by blocking exportin 1(XPO1)

description: A member of the class of triazoles that is 1H-1,2,4-triazole substituted by (1Z)-3-oxo-3-[2-(pyrazin-2-yl)hydrazinyl]prop-1-en-1-yl and 3,5-bis(trifluoromethyl)phenyl groups at positions 1 and 3, respectively. It is a prescription medicine approved for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma.


pexidartinib

mrdef: Pexidartinib is a small molecule tyrosine kinase inhibitor that targets colony stimulating factor 1 receptor (CSF1R), KIT proto-oncogene receptor tyrosine kinase (KIT), and FMS-like tyrosine kinase 3 (FLT3) harboring an internal tandem duplication (ITD) mutation. Overexpression of the CSF1R ligand promotes cell proliferation and accumulation in the synovium. In vitro, pexidartinib inhibited proliferation of cell lines dependent on CSF1R and ligand-induced autophosphorylation of CSF1R. Pexidartinib also inhibited the proliferation of a CSF1R dependent cell line in vivo.

description: A pyrrolopyridine that is 5-chloro-1H-pyrrolo[2,3-b]pyridine which is substituted by a [6-({[6-(trifluoromethyl)pyridin-3-yl]methyl}amino)pyridin-3-yl]methyl group at position 3. It is a potent multi-targeted receptor tyrosine kinase inhibitor of CSF-1R, KIT, and FLT3 (IC50 of 20 nM, 10 nM and 160 nM, respectively). Approved by the FDA for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT).


tenosynovial giant cell tumor

description: A tumor usually arising in the synovium of joints, bursa or tendon sheath. It is characterized by the presence of mononuclear cells, multinucleated osteoclast-like giant cells, hemosiderin-laden macrophages, foam cells, and an inflammatory infiltrate. According to the growth pattern, it is classified as localized or diffuse.


pretomanid

mrdef: Pretomanid Tablet is a nitroimidazooxazine antimycobacterial drug. Pretomanid kills actively replicating M. tuberculosis by inhibiting mycolic acid biosynthesis, thereby blocking cell wall production. Under anaerobic conditions, against non-replicating bacteria, pretomanid acts as a respiratory poison following nitric oxide release


extensively drug-resistant tuberculosis

description: A type of drug-resistant tuberculosis that is resistant to any fluoroquinolone, and at least one of three second-line injectable drugs (capreomycin, kanamycin, and amikacin), in addition to resistance to rifampicin and isoniazid.


entrectinib

mrdef: Entrectinib is an inhibitor of the tropomyosin receptor tyrosine kinases (TRK) TRKA, TRKB, and TRKC (encoded by the neurotrophic tyrosine receptor kinase [NTRK] genes NTRK1, NTRK2, and NTRK3, respectively), proto-oncogene tyrosine-protein kinase ROS1 (ROS1), and anaplastic lymphoma kinase (ALK).

description: A member of the class of indazoles that is 1H-indazole substituted by [4-(4-methylpiperazin-1-yl)-2-(tetrahydro-2H-pyran-4-ylamino)benzoyl]amino and 3,5-difluorobenzyl groups at positions 3 and 5, respectively. It is a potent inhibitor of TRKA, TRKB, TRKC, ROS1, and ALK (IC50 values of 0.1 to 1.7 nM), and used for the treatment of NTRK, ROS1 and ALK gene fusion-positive solid tumours.


name: Reactive oxygen species 1 positive non-small cell lung cancer


upadacitinib

mrdef: Upadacitinib is a Janus kinase (JAK) inhibitor. JAKs are intracellular enzymes which transmit signals arising from cytokine or growth factor-receptor interactions on the cellular membrane to influence cellular processes of hematopoiesis and immune cell function. Within the signaling pathway, JAKs phosphorylate and activate Signal Transducers and Activators of Transcription (STATs) which modulate intracellular activity including gene expression. Upadacitinib modulates the signaling pathway at the point of JAKs, preventing the phosphorylation and activation of STATs.


fedratinib

mrdef: Fedratinib is an oral kinase inhibitor with activity against wild type and mutationally activated Janus Associated Kinase 2 (JAK2) and FMS-like tyrosine kinase 3 (FLT3). Fedratinib is a JAK2-selective inhibitor with higher inhibitory activity for JAK2 over family members JAK1, JAK3 and TYK2. Abnormal activation of JAK2 is associated with myeloproliferative neoplasms (MPNs), including myelofibrosis and polycythemia vera.


lefamulin

mrdef: XENLETA is a semi-synthetic antibacterial agent for oral and intravenous administration. Lefamulin inhibits bacterial protein synthesis through interactions (hydrogen bonds, hydrophobic interactions, and Van der Waals forces) with the A- and P-sites of the peptidyl transferase center (PTC) in domain V of the 23s rRNA of the 50S subunit. The binding pocket of the bacterial ribosome closes around the mutilin core for an induced fit that prevents correct positioning of tRNA.


gallium (68Ga) edotreotide

mrdef: A radioconjugate consisting of the octreotide derivative edotreotide labeled with gallium 68 (Ga-68) with potential application in somatostatin receptor (SSTR) imaging in conjunction with positron emission tomography (PET). Similar to octreotide, gallium Ga 68-edotreotide binds to SSTRs, especially type 2 receptors, present on the cell membranes of many types of neuroendocrine tumor cells and their metastases, thereby allowing for imaging of SSTR-expressing cells upon PET. Gallium Ga 68-edotreotide is produced by substituting tyrosine for phenylalanine at the 3 position of the somatostatin analogue octreotide (Tyr3-octreotide or TOC) and chelating the substituted octreotide to Ga-68 via the macrocyclic chelating agent dodecanetetraacetic acid (DOTA).


name: Positron emission tomography with computed tomography of whole body using gallium (68-Ga) dotatate


tenapanor

mrdef: IBSRELA (tenapanor) tablets contain tenapanor hydrochloride as an active ingredient. Tenapanor hydrochloride is a sodium/hydrogen exchanger 3 (NHE3) inhibitor for oral use, an antiporter expressed on the apical surface of the small intestine and colon primarily responsible for the absorption of dietary sodium. In vitro and animal studies indicate its major metabolite, M1, is not active against NHE3. By inhibiting NHE3 on the apical surface of the enterocytes, tenapanor reduces absorption of sodium from the small intestine and colon, resulting in an increase in water secretion into the intestinal lumen, which accelerates intestinal transit time and results in a softer stool consistency.


trifarotene

mrdef: AKLIEF Cream for topical administration contains 0.005% (50 mcg/g) trifarotene. Trifarotene is a terphenyl acid derivative and is a retinoid. Trifarotene is an agonist of retinoic acid receptors (RAR), with particular activity at the gamma subtype of RAR. Stimulation of RAR results in modulation of target genes which are associated with various processes, including cell differentiation and mediation of inflammation. The exact process by which trifarotene ameliorates acne is unknown.


lasmiditan

mrdef: Reyvow (lasmiditan) is a serotonin (5-HT) 1F receptor agonist for oral administration, bindinding with high affinity to the 5-HT1F receptor. Lasmiditan presumably exerts its therapeutic effects in the treatment of migraine through agonist effects at the 5-HT1F receptor; however, the precise mechanism is unknown.


cefiderocol

mrdef: Cefiderocol is a cephalosporin antibacterial with activity against Gram-negative aerobic bacteria. Cefiderocol functions as a siderophore and binds to extracellular free ferric iron. In addition to passive diffusion via porin channels, cefiderocol is actively transported across the outer cell membrane of bacteria into the periplasmic space using a siderophore iron uptake mechanism. Cefiderocol exerts bactericidal action by inhibiting cell wall biosynthesis through binding to penicillin-binding proteins (PBPs)

description: A fourth-generation siderophore cephalosporin antibiotic having {1-[2-(2-chloro-3,4-dihydroxybenzamido)ethyl]pyrrolidinium-1-yl}methyl and [(2Z)-2-(2-amino-1,3-thiazol-4-yl)-2-{[(2-carboxypropan-2-yl)oxy]imino}acetyl]amino side groups located at positions 3 and 7 respectively, developed to combat a variety of bacterial pathogens, including beta-lactam- and carbapenem-resistant organisms.


zanubrutinib

mrdef: BRUKINSA (zanubrutinib) is a Bruton’s tyrosine kinase (BTK) inhibitor. Zanubrutinib forms a covalent bond with a cysteine residue in the BTK active site, leading to inhibition of BTK activity. BTK is a signaling molecule of the B-cell antigen receptor (BCR) and cytokine receptor pathways. In B-cells, BTK signaling results in activation of pathways necessary for B-cell proliferation, trafficking, chemotaxis, and adhesion. In nonclinical studies, zanubrutinib inhibited malignant B-cell proliferation and reduced tumor growth.


cenobamate

mrdef: The precise mechanism by which cenobamate exerts its therapeutic effects in patients with partial-onset seizures is unknown. Cenobamate has been demonstrated to reduce repetitive neuronal firing by inhibiting voltage-gated sodium  currents. It is also a positive allosteric modulator of the gamma-aminobutyric acid (GABAA) ion channel.


voxelotor

mrdef: Voxelotor is a hemoglobin S (HbS) polymerization inhibitor that binds to HbS with a 1:1 stoichiometry and exhibits preferential partitioning to red blood cells (RBCs). By increasing the affinity of Hb for oxygen, voxelotor demonstrates dose-dependent inhibition of HbS polymerization. Nonclinical studies suggest that voxelotor may inhibit RBC sickling, improve RBC deformability, and reduce whole blood viscosity.


golodirsen

mrdef: Golodirsen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass. It is designed to bind to exon 53 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon 53 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping.


lumateperone

mrdef: The mechanism of action of lumateperone in the treatment of schizophrenia is unknown. However, the efficacy of lumateperone could be mediated through a combination of antagonist activity at central serotonin 5-HT2A receptors and postsynaptic antagonist activity at central dopamine D2 receptors.


lemborexant

mrdef: The mechanism of action of lemborexant in the treatment of insomnia is presumed to be through antagonism of orexin receptors. The orexin neuropeptide signaling system plays a role in wakefulness. Blocking the binding of wake-promoting neuropeptides orexin A and orexin B to receptors OX1R and OX2R is thought to suppress wake drive.


ubrogepant

mrdef: Ubrogepant is a calcitonin gene-related peptide receptor antagonist.