PEGLOTICASE

a recombinant uricase and achieves its therapeutic effect by catalyzing the oxidation of uric acid to allantoin, thereby lowering serum uric acid


dermatomycosis

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Superficial infections of the skin or its appendages by any of various fungi.


Clidinium

a synthetic anticholinergic agent that has antispasmodic and antisecretory effect on the gastrointestinal tract


enterocolitis

An inflammatory process affecting the small intestine and colon. Causes include viruses, bacteria, radiation, and antibiotics use.


Encainide

One of the ANTI-ARRHYTHMIA AGENTS, it blocks VOLTAGE-GATED SODIUM CHANNELS and slows conduction within the His-Purkinje system and MYOCARDIUM.

4-Methoxy-N-phenylbenzamide in which the hydrogen at the 2 position of the phenyl group is substituted by a 2-(1-methylpiperidin-2-yl)ethyl group. A class Ic antiarrhythmic, the hydrochloride was used for the treatment of severe or life-threatening ventricular arrhythmias, but it was associated with increased death rates in patients who had asymptomatic heart rhythm abnormalities after a recent heart attack and was withdrawn from the market.


Premature ventricular contraction

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Premature ventricular contractions (PVC) or ventricular extrasystoles are premature contractions of the heart that arise in response to an impulse in the ventricles rather than the normal impulse from the sinoatrial (SA) node.


Posaconazole

an antifungal medicine that belongs to the triazole group, it works by preventing the formation of ergosterol, which is an important part of fungal cell walls

An N-arylpiperazine that consists of piperazine carrying two 4-substituted phenyl groups at positions 1 and 4. A triazole antifungal drug.


aspergillosis

Aspergillosis is an infection, growth, or allergic response caused by the Aspergillus fungus. There are several different kinds of aspergillosis. One kind is allergic bronchopulmonary aspergillosis (also called ABPA), a condition where the fungus causes allergic respiratory symptoms similar to asthma, such as wheezing and coughing, but does not actually invade and destroy tissue. Another kind of aspergillosis is invasive aspergillosis. This infection usually affects people with weakened immune systems due to cancer, AIDS, leukemia, organ transplantation, chemotherapy, or other conditions or events that reduce the number of normal white blood cells. In this condition, the fungus invades and damages tissues in the body. Invasive aspergillosis most commonly affects the lungs, but can also cause infection in many other organs and can spread throughout the body (commonly affecting the kidneys and brain). Aspergilloma, a growth (fungus ball) that develops in an area of previous lung disease such as tuberculosis or lung abscess, is a third kind of aspergillosis. This type of aspergillosis is composed of a tangled mass of fungus fibers, blood clots, and white blood cells. The fungus ball gradually enlarges, destroying lung tissue in the process, but usually does not spread to other areas.


fusariosis

Fusariosis describes a superficial, locally invasive, disseminated infection with the pathogenic fungus species, Fusarium, often found in soil and water, which is mainly transmitted to humans through traumatic inoculation and that manifests with keratitis, onychomycosis and less frequently peritonitis and cellulitis. In the immunocompromised, disseminated fusariosis is more common and it manifests with refractory fever, skin lesions (ecthyma-like, target, and multiple subcutaneous nodules), severe myalgias and sino-pulmonary infections.


chromomycosis

Chromomycosis is a chronic cutaneous and subcutaneous fungal infection, found mainly in subtropical and tropical areas (in soil and plant debris and transmitted by traumatic inoculation), and characterized clinically by slow growing, verrucous nodules, squamous plaques, or chronic limited lesions which are most commonly found on the lower limbs and which are characterized histologically by the presence of muriform cells. It is caused by dematiaceous fungi, with the main etiological agents being Fonsecaea pedrosoi, Phialophora verrucosa and Cladophialophora carrionii. Rarely, it can be caused by Rhinocladiella aquaspersa.


Oxyphenisatin acetate

A laxative that undergoes enterohepatic circulation. It may cause jaundice.


PEGINTERFERON BETA-1A

an interferon beta-1a to which a single, linear 20,000 dalton methoxy poly(ethyleneglycol)-O-2-methylpropionaldehyde molecule is covalently attached to the alpha amino group of the N-terminal amino acid residue. A possible role for IFNs in prophylaxis or early treatment of COVID-19 has been suggested to compensate for possibly insufficient endogenous IFN production. However, the efficacy and safety of IFNs for treatment or prevention of COVID-19 were not established.


INTERFERON BETA-1A

An interferon beta-1 subtype that has a methionine at position 1, a cysteine at position 17, and is glycosylated at position 80. It functions as an ANTI-VIRAL AGENT and IMMUNOMODULATOR and is used to manage the symptoms of RELAPSING-REMITTING MULTIPLE SCLEROSIS. A possible role for IFNs in prophylaxis or early treatment of COVID-19 has been suggested to compensate for possibly insufficient endogenous IFN production. However, the efficacy and safety of IFNs for treatment or prevention of COVID-19 were not established.


INTERFERON BETA-1B

A non-glycosylated form of interferon beta-1 that has a serine at position 17. It is used in the treatment of both RELAPSING-REMITTING MULTIPLE SCLEROSIS and CHRONIC PROGRESSIVE MULTIPLE SCLEROSIS. A possible role for IFNs in prophylaxis or early treatment of COVID-19 has been suggested to compensate for possibly insufficient endogenous IFN production. However, the efficacy and safety of IFNs for treatment or prevention of COVID-19 were not established.


Edaravone

Edaravone protects neurons from oxidative stress by scavenging free radicals that may cause cellular damage, and this mechanism is expected to be effective both in patients with acute cerebral infarction and in those with ALS

A pyrazolone that is 2,4-dihydro-3H-pyrazol-3-one which is substituted at positions 2 and 5 by phenyl and methyl groups, respectively.


A proteolytic enzyme in the serine protease family found in many tissues which converts PLASMINOGEN to FIBRINOLYSIN. It has fibrin-binding activity and is immunologically different from UROKINASE-TYPE PLASMINOGEN ACTIVATOR. The primary sequence, composed of 527 amino acids, is identical in both the naturally occurring and synthetic proteases. Is currently clinical tested as  treatment for COVID-19 patients.


acute myocardial infarction

Necrosis of the myocardium, as a result of interruption of the blood supply to the area. It is characterized by a severe and rapid onset of symptoms that may include chest pain, often radiating to the left arm and left side of the neck, dyspnea, sweating, and palpitations.


Cabozantinib

Cabozantinib inhibits the tyrosine kinase activity of MET, VEGFR-1, -2 and -3, AXL, RET, ROS1, TYRO3, MER, KIT, TRKB, FLT-3, and TIE-2. These receptor tyrosine kinases are involved in both normal cellular function and pathologic processes such as oncogenesis, metastasis, tumor angiogenesis, drug resistance, and maintenance of the tumor microenvironment.

A dicarboxylic acid diamide that is N-phenyl-N'-(4-fluorophenyl)cyclopropane-1,1-dicarboxamide in which the hydrogen at position 4 on the phenyl ring is substituted by a (6,7-dimethoxyquinolin-4-yl)oxy group. A multi-tyrosine kinase inhibitor, used (as its malate salt) for the treatment of progressive, metastatic, medullary thyroid cancer.


Iron supplement therapy

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Opicapone

a peripheral, selective and reversible catechol-O-methyltransferase (COMT) inhibitor endowed with a high binding affinity (sub-picomolar) that translates into a slow complex dissociation rate constant and a long duration of action (>24 hours) in vivo


Gluconolactone

An  aldono-1,5-lactone obtained from D-gluconic acid.


bladder calculus

A concretion in the urinary bladder.


Faropenem daloxate

a faropenem prodrug for treating bacterial infections


FERUMOXSIL

a large superparamagnetic iron oxide colloid; a miscible darkening agent; see also the dextran-coated superparamagnetic iron oxides - ferumoxides and ferumoxtran


PORACTANT ALFA

Endogenous pulmonary surfactant (isolated from porcine lungs) reduces surface tension at the air-liquid interface of the alveoli during ventilation and stabilizes the alveoli against collapse at resting transpulmonary pressures. A deficiency of pulmonary surfactant in preterm infants results in Respiratory Distress Syndrome (RDS) characterized by poor lung expansion, inadequate gas exchange, and a gradual collapse of the lungs (atelectasis).


newborn respiratory distress syndrome

A condition beginning in the first day of life that results from inadequate surfactant production, causing increased work of breathing and impaired gas exchange.


Talipexole

dopamine receptor agonist; structure given in first source


Venetoclax

Venetoclax is a selective and orally bioavailable small-molecule inhibitor of BCL-2, an anti-apoptotic protein. Overexpression of BCL-2 has been demonstrated in CLL cells where it mediates tumor cell survival and has been associated with resistance to chemotherapeutics. Venetoclax helps restore the process of apoptosis by binding directly to the BCL-2 protein, displacing pro-apoptotic proteins like BIM, triggering mitochondrial outer membrane permeabilization and the activation of caspases. In nonclinical studies, venetoclax has demonstrated cytotoxic activity in tumor cells that overexpress BCL-2.

A member of the class of pyrrolopyridines that is a potent inhibitor of the antiapoptotic protein B-cell lymphoma 2. It is used for treamtment of chronic lymphocytic leukemia with 17p deletion.


B-cell chronic lymphocytic leukemia

B-cell chronic lymphocytic leukemia (B-CLL) is a type of B-cell non-Hodgkin lymphoma, and the most common form of leukemia in Western countries, affecting elderly adults (mean age of 67 and 72 years) with a slight male predominance (1.7:1), and characterized by a highly variable clinical presentation that can include asymptomatic disease or non-specific B-symptoms such as unintentional weight loss, severe fatigue, fever (without evidence of infection), and night sweats as well as cervical lymphadenopathy, splenomegaly and frequent infections. Some patients can also develop autoimmune complications such as autoimmune hemolytic anemia or immune thrombocytopenia. The clinical course is extremely heterogeneous with survival ranging from a few months to several decades.


factor XIII, A subunit, deficiency of

Deficiency of factor XIII subunit A, leading to a reduced factor XIII activity. Activated Factor XIII cross-links fibrin polymers solidifying the clot.


DORNASE ALFA

A recombinant human deoxyribonuclease I (rhDNAse) with selective DNA cleaving activity. Upon intrapleural administration, dornase alfa catalyzes the degradation of extracellular DNA in airway secretions, which can reduce their viscosity. Thus, dornase alfa may both promote the clearing of airway mucus and improve pulmonary function.


cystic fibrosis

Cystic fibrosis (CF) is a genetic disorder characterized by the production of sweat with a high salt content and mucus secretions with an abnormal viscosity.


Lofexidine

Lofexidine is a central alpha-2 adrenergic agonist that binds to receptors on adrenergic neurons. This reduces the release of norepinephrine and decreases sympathetic tone.


Eribulin

Eribulin inhibits the growth phase of microtubules without affecting the shortening phase and sequesters tubulin into nonproductive aggregates. Eribulin exerts its effects via a tubulin-based antimitotic mechanism leading to G2/M cell-cycle block, disruption of mitotic spindles, and, ultimately, apoptotic cell death after prolonged mitotic blockage. In addition, eribulin treatment of human breast cancer cells caused changes in morphology and gene expression as well as decreased migration and invasiveness in vitro. In mouse xenograft models of human breast cancer, eribulin treatment was associated with increased vascular perfusion and permeability in the tumor cores, resulting in reduced tumor hypoxia, and changes in the expression of genes in tumor specimens associated with a change in phenotype.

A fully synthetic macrocyclic ketone analogue of marine sponge natural products. Inhibits growth phase of microtubules via tubulin-based antimitotic mechanism, which leads to G2/M cell-cycle block, disruption of mitotic spindles, and, ultimately, apoptotic cell death after prolonged mitotic blockage


liposarcoma

A usually painless malignant tumor that arises from adipose tissue. Microscopically, it may contain a spectrum of neoplastic adipocytes ranging from lipoblasts to pleomorphic malignant adipocytes. Morphologic variants include: well differentiated, dedifferentiated, pleomorphic, and myxoid liposarcoma. The metastatic potential is higher in less differentiated tumors.


covalent attachment of PEG to enzyme produces active, stable complex; non-immunogenic; used in treatment of leukemia


acute lymphoblastic leukemia

Leukemia with an acute onset, characterized by the presence of lymphoblasts in the bone marrow and the peripheral blood. It includes the acute B lymphoblastic leukemia and acute T lymphoblastic leukemia.


Elbasvir

inhibits NS5A protein of hepatitis C virus

A complex organic heterotetracyclic compound that is a hepatitis C virus nonstructural protein 5A inhibitor used in combination with grazoprevir (under the brand name Zepatier) for treatment of chronic HCV genotypes 1 or 4 infection in adults.


Grazoprevir

An azamacrocyclic compound that is a hepatitis C protease inhibitor used in combination with elbasvir (under the brand name Zepatier) for treatment of chronic HCV genotypes 1 or 4 infection in adults.


Oxetorona

treatment of migraine; RN given refers to parent cpd; structure


Artemisinin

antimalarial from Chinese medicinal herb Artemisia annua

A sesquiterpene lactone obtained from sweet wormwood, Artemisia annua, which is used as an antimalarial for the treatment of multi-drug resistant strains of falciparum malaria.


Plasmodium falciparum malaria

Malaria resulting from infection by Plasmodium falciparum.


IXEKIZUMAB

A humanized anti-interleukin 17 monoclonal antibody used for the treatment of PSORIASIS.


EFTRENONACOG ALFA

consists of factor IX fused to the Fc fragment of human IgG1


Acute Pain

A sensation of discomfort or distress that has a severe or rapid onset.


BESILESOMAB

a mouse monoclonal antibody labelled with the radioactive isotope technetium-99m for the detection of septic arthroplasty


Racecadotril

A potent inhibitor of membrane metalloendopeptidase (ENKEPHALINASE). Thiorphan potentiates morphine-induced ANALGESIA and attenuates naloxone-precipitated withdrawal symptoms.


acute diarrhea

Acute form of diarrhea.


Fasudil

An isoquinoline substituted by a (1,4-diazepan-1-yl)sulfonyl group at position 5. It is a Rho-kinase inhibitor and its hydrochloride hydrate form is approved for the treatment of cerebral vasospasm and cerebral ischemia.


brain ischemia

Diminished or absent blood supply to the brain caused by obstruction (thrombosis or embolism) of an artery resulting in neurologic damage.


DENILEUKIN DIFTITOX

more potent, truncated form of DAB(486)-IL-2; inhibit HIV-1 RNA production in infected cells


primary cutaneous T-cell non-Hodgkin lymphoma

A T-cell non-Hodgkin lymphoma arising from the skin. Representative examples include mycosis fungoides and primary cutaneous anaplastic large cell lymphoma.


ALEFACEPT

A recombinant human fusion protein of LFA-3 antigen and immunoglobulin IGG1 that binds to CD2 ANTIGENS on memory T-LYMPHOCYTES, preventing their activation and proliferation. It is used in the management of moderate to severe chronic plaque PSORIASIS.


ABATACEPT

A fusion protein immunoconjugate of the extracellular domain of CTLA4 and the Fc domain of human IgG1. It functions as a T-cell co-stimulation blocker that inhibits TNF-ALPHA and prevents the activation of T-LYMPHOCYTES. It is used in the treatment of RHEUMATOID ARTHRITIS.


juvenile idiopathic arthritis

Juvenile idiopathic arthritis (JIA) is the term used to describe a group of inflammatory articular disorders of unknown cause that begin before the age of 16 and last over 6 weeks. The term juvenile idiopathic arthritis was chosen to signify the absence of any known mechanism underlying the disorder and to highlight the necessity of excluding other types of arthritis occurring in well defined diseases (in particular arthritis occurring in association with infectious, inflammatory and haematooncologic diseases).


Nafamostat

inhibitor of trypsin, plasmin, pancreatic kallikrein, plasma kallikrein & thrombin; strongly inhibits esterolytic activities of C1r & C1 esterase complement-mediated hemolysis; antineoplastic; RN given refers to parent cpd


Benoxaprofen

A monocarboxylic acid that is propionic acid substituted at position 2 by a 2-(4-chlorophenyl)-1,3-benzoxazol-5-yl group. It was used as a non-steroidal anti-inflammatory drug until 1982 when it was withdrawn from the market due to adverse side-effects including liver necrosis, photosensitivity, and carcinogenicity in animals.


Flunoxaprofen

A monocarboxylic acid that is propionic acid substituted at position 2 by a 2-(4-chlorophenyl)-1,3-benzoxazol-5-yl group (the S-enantiomer). Although it was shown to be effective in treatment of rheumatoid arthritis and osteoarthritis, the clinical use of flunoxaprofen was discontinued due to possible hepatotoxic side-effects.


Climbazole

Climbazole is a conazole fungicide. It is also use in shampoo and cleaner as an anti-dandruff agents and anti-bacteria agent.

A racemate composed of equimolar amounts of (R)- and (S)-climbazole. It is a topically applied antifungal agent used to treat human fungal skin infections.


seborrheic dermatitis

A chronic, inflammatory skin disorder that affects the scalp, central face and skin folds; it is characterized by scaling and itching.


TENECTEPLASE

A tissue plasminogen activator enzyme that acts as a fibrinolytic agent; it is used for the dissolution of blood clots, such as those that occur in acute MYOCARDIAL INFARCTION.


Ixazomib

an oral proteasome inhibitor with antineoplastic activity; MLN2238 is the biologically active form of MLN9708

A glycine derivative that is the amide obtained by formal condensation of the carboxy group of N-(2,5-dichlorobenzoyl)glycine with the amino group of [(1R)-1-amino-3-methylbutyl]boronic acid. The active metabolite of ixazomib citrate, it is used in combination therapy for treatment of multiple myeloma.


plasma cell myeloma

A bone marrow-based plasma cell neoplasm characterized by a serum monoclonal protein and skeletal destruction with osteolytic lesions, pathological fractures, bone pain, hypercalcemia, and anemia. Clinical variants include non-secretory myeloma, smoldering myeloma, indolent myeloma, and plasma cell leukemia. (WHO, 2001)


Brivaracetam

Brivaracetam displays a high and selective affinity for synaptic vesicle protein 2A (SV2A) in the brain, which may contribute to the anticonvulsant effect.

A non-proteinogenic amino acid derivative that is butanamide in which the pro-S hydrogen at position 2 is replaced by a (4R)-2-oxo-4-propylpyrrolidin-1-yl. Used for treatment of partial onset seizures related to epilepsy.


focal epilepsy

A seizure caused by a localized disorder.


DARATUMUMAB

Daratumumab is an IgG1k human monoclonal antibody (mAb) that binds to CD38 and inhibits the growth of CD38 expressing tumor cells by inducing apoptosis directly through Fc mediated cross linking as well as by immune-mediated tumor cell lysis through complement dependent cytotoxicity (CDC), antibody dependent cell mediated cytotoxicity (ADCC) and antibody dependent cellular phagocytosis (ADCP).


SUSOCTOCOG ALFA

a recombinant B-domain deleted porcine sequence Factor VIII, binds to von Willebrand factor


acquired hemophilia A

An acquired form of hemophilia A, resulting in spontaneous bleeding in individuals with no history of bleeding disorders. It is believed to be caused by spontaneous inhibition of clotting factor VIII by autoantibodies, and is usually associated with other autoimmune conditions.


Asparaginate

A hydrolase enzyme that converts L-asparagine and water to L-aspartate and NH3. EC 3.5.1.1.

An alpha-amino-acid anion that is the conjugate base of asparagine, arising from deprotonation of the carboxy group.


NIVOLUMAB

A genetically engineered, fully humanized immunoglobulin G4 monoclonal antibody that binds to the PD-1 RECEPTOR, activating an immune response to tumor cells. It is used as monotherapy or in combination with IPILIMUMAB for the treatment of advanced malignant MELANOMA. It is also used in the treatment of advanced or recurring NON-SMALL CELL LUNG CANCER; RENAL CELL CARCINOMA; and HODGKIN'S LYMPHOMA.


NECITUMUMAB

a fully human antibody that has similar antitumor potency as the chimeric cetuximab/Erbitux and might represent a safer therapeutic alternative in EGF receptor inhibition


primary cerebellar degeneration

A heterogenous group of degenerative syndromes marked by progressive cerebellar dysfunction either in isolation or combined with other neurologic manifestations. Sporadic and inherited subtypes occur. Inheritance patterns include autosomal dominant, autosomal recessive, and X-linked.


ADALIMUMAB

A humanized monoclonal antibody that binds specifically to TNF-ALPHA and blocks its interaction with endogenous TNF RECEPTORS to modulate INFLAMMATION. It is used in the treatment of RHEUMATOID ARTHRITIS; PSORIATIC ARTHRITIS; CROHN'S DISEASE and ULCERATIVE COLITIS.


hidradenitis suppurativa

A chronic suppurative and cicatricial disease of the apocrine glands occurring chiefly in the axillae in women and in the groin and anal regions in men. It is characterized by poral occlusion with secondary bacterial infection, evolving into abscesses which eventually rupture. As the disease becomes chronic, ulcers appear, sinus tracts enlarge, fistulas develop, and fibrosis and scarring become evident.


ELOTUZUMAB

a humanized monoclonal antibody that selectively targets glycoproteins expressed on myeloma cells


Felbinac

an anti-inflammatory/analgesic agent which has been developed into a topical gel for local treatment and pain and inflammation associated with conditions of the musculo-skeletal system

A monocarboxylic acid in which one of the alpha-hydrogens is substituted by a biphenyl-4-yl group. An active metabolite of fenbufen, it is used as a topical medicine to treat muscle inflammation and arthritis.


Echothiophate

A potent, long-acting cholinesterase inhibitor used as a miotic in the treatment of glaucoma.

The phosphorothioate obtained by formal condensation of diethyl phosphate with N,N,N-trimethyl-2-sulfanylethanaminium. An irreversible acetylcholinesterase inhibitor, its iodide salt is used an ocular antihypertensive in the treatment of open-angle glaucoma, particularly when other drugs have proved inadequate.


Stargardt disease

Stargardt disease, also known as Stargardt 1 (STGD1), is an autosomal recessive form of retinal dystrophy that is usually characterized by a progressive loss of central vision associated with irregular macular and perimacular yellow-white fundus flecks, and a so-called ''beaten bronze'' atrophic central macular lesion.


Lorcaserin

a serotonin 2C receptor agonist for oral administration used for chronic weight management

A benzazepine that is 2,3,4,5-tetrahydro-3-benzazepine substituted at position 1 by a methyl group and a t position 6 by a chloro group.


obesity disorder

A disorder involving an excessive amount of body fat.


Uridine triacetate is an acetylated form of uridine. Following oral administration, uridine triacetate is deacetylated by nonspecific esterases present throughout the body, yielding uridine in the systemic circulation of patients with hereditary orotic aciduria who cannot synthesize adequate quantities of uridine due to a genetic defect in uridine nucleotide synthesis.


Lesinurad

Lesinurad reduces serum uric acid levels by inhibiting the function of transporter proteins involved in uric acid reabsorption in the kidney. Lesinurad inhibited the function of two apical transporters responsible for uric acid reabsorption, uric acid transporter 1 (URAT1) and organic anion transporter 4 (OAT4), with IC50 values of 7.3 and 3.7 μM, respectively. URAT1 is responsible for the majority of the reabsorption of filtered uric acid from the renal tubular lumen. OAT4 is a uric acid transporter associated with diuretic-induced hyperuricemia.

A member of the class of triazoles that is [(3-bromo-1,2,4-triazol-5-yl)sulfanyl]acetic acid substituted at position 1 of the triazole ring by a 4-cyclopropylnaphthalen-1-yl group. Used for treatment of gout.


Alclofenac

Alclofenac is a non-steroidal anti-inflammatory agent, withdrawn from the market. It acts by blocking the synthesis of prostaglandins through the inhibition of cyclooxygenase, which converts arachidonic acid to cyclic endoperoxides, precursors of prostaglandins. Inhibition of prostaglandin synthesis accounts for their analgesic, antipyretic, and platelet-inhibitory actions.

An aromatic ether in which the ether oxygen links an allyl group to the 4-position of (3-chlorophenyl)acetic acid.A non-steroidal anti-inflammatory drug, it was withdrawn from the UK market in 1979 due to concerns with its association with vasculitis and rash.


Cobimetinib

Cobimetinib is a reversible inhibitor of mitogen-activated protein kinase (MAPK)/extracellular signal regulated kinase 1 (MEK1) and MEK2. MEK proteins are upstream regulators of the extracellular signal-related kinase (ERK) pathway, which promotes cellular proliferation. BRAF V600E and K mutations result in constitutive activation of the BRAF pathway which includes MEK1 and MEK2. In mice implanted with tumor cell lines expressing BRAF V600E, cobimetinib inhibited tumor cell growth.

A member of the class of N-acylazetidines obtained by selective formal condensation of the carboxy group of 3,4-difluoro-2-(2-fluoro-4-iodoanilino)benzoic acid with the secondary amino group from the azetidine ring of 3-[(2S)-piperidin-2-yl]azetidin-3-ol. An inhibitor of mitogen-activated protein kinase that is used (as its fumarate salt) in combination with vemurafenib for the treatment of patients with unresectable or metastatic melanoma.


Aprindine

A class Ib anti-arrhythmia agent used to manage ventricular and supraventricular arrhythmias.


Supraventricular arrhythmia

A type of arrhythmia that originates above the ventricles, whereby the electrical impulse propagates down the normal His Purkinje system similar to normal sinus rhythm.


Ventricular arrhythmia

A disorder characterized by an electrocardiographic finding of an atypical cardiac rhythm resulting from a pathologic process in the cardiac ventricles.


epilepsy

A brain disorder characterized by episodes of abnormally increased neuronal discharge resulting in transient episodes of sensory or motor neurological dysfunction, or psychic dysfunction. These episodes may or may not be associated with loss of consciousness or convulsions.


Saroglitazar

A monocarboxylic acid that is (2S)-2-ethoxy-3-(p-ethoxyphenyl)propanoic acid in which one of the methyl hydrogens of the p-ethoxy substituent has been replaced by the nitrogen of 2-methyl-5-[4-(methylthio)phenyl]-1H-pyrrole. An agonist at the subtypes alpha and gamma of the peroxisome proliferator-activated receptor (PPAR) with predominant PPARalpha activity, it is used in the treatment of type 2 diabetes.


Dyslipidemia due to type 2 diabetes mellitus


Pitolisant

Pitolisant is a potent, orally active histamine H3-receptor antagonist/inverse agonist which, via its blockade of histamine auto-receptors enhances the activity of brain histaminergic neurons, a major arousal system with widespread projections to the whole brain. Pitolisant also modulates various neurotransmitter systems, increasing acetylcholine, noradrenaline and dopamine release in the brain.


narcolepsy

A sleep disorder characterized by a tendency for excessive sleepiness during the day which occurs even after adequate sleep in the nighttime. The persons who suffer from this condition experience fatigue and may fall asleep at inappropriate times during the day.


Alkaline phosphatase

An enzyme that catalyzes the conversion of an orthophosphoric monoester and water to an alcohol and orthophosphate. EC 3.1.3.1.


hypophosphatasia

Hypophosphatasia (HPP) is a rare heritable metabolic disorder characterized by defective mineralization of bone and/or teeth in the presence of reduced activity of unfractionated serum alkaline phosphatase (ALP). The clinical spectrum is extremely wide, from stillbirth at one end to fractures of the lower extremities in adulthood, at the other, or even no bone manifestations (odontohypophosphatasia).


ASTODRIMER

microbicide, polyanion-based entry inhibitor, astodrimer is a broad-spectrum dendrimer consisting of a divalent benzhydrylamine core and four layers of lysine branches


purified collagenase clostridium histolyticum, consisting of two microbial collagenases in a defined mass ratio, Collagenase AUX-I and Collagenase AUX-II, which are isolated and purified from the fermentation of Clostridium histolyticum bacteria


Peyronie disease

A condition characterized by hardening of the penis due to the formation of fibrous plaques on the dorsolateral aspect of the penis, usually involving the membrane (tunica albuginea) surrounding the erectile tissue (corpus cavernosum penis). This may eventually cause a painful deformity of the shaft or constriction of the urethra, or both.


Sufentanil

An opioid analgesic that is used as an adjunct in anesthesia, in balanced anesthesia, and as a primary anesthetic agent.

An anilide resulting from the formal condensation of the aryl amino group of 4-(methoxymethyl)-N-phenyl-1-[2-(2-thienyl)ethyl]piperidin-4-amine with propanoic acid.


Post-Operative Pain

A sensation of discomfort secondary to surgery.


PEMBROLIZUMAB

A humanized monoclonal immunoglobulin (Ig) G4 antibody directed against human cell surface receptor PD-1 (programmed death-1 or programmed cell death-1) with potential immune checkpoint inhibitory and antineoplastic activities. Upon administration, pembrolizumab binds to PD-1, an inhibitory signaling receptor expressed on the surface of activated T cells, and blocks the binding to and activation of PD-1 by its ligands, which results in the activation of T-cell-mediated immune responses against tumor cells. The ligands for PD-1 include programmed cell death ligand 1 (PD-L1), overexpressed on certain cancer cells, and programmed cell death ligand 2 (PD-L2), which is primarily expressed on APCs. Activated PD-1 negatively regulates T-cell activation and plays a key role in in tumor evasion from host immunity.


Celiprolol

A cardioselective beta-1 adrenergic antagonist that has intrinsic symopathomimetic activity. It is used in the management of ANGINA PECTORIS and HYPERTENSION.


Isoxsuprine

A beta-adrenergic agonist that causes direct relaxation of uterine and vascular smooth muscle. Its vasodilating actions are greater on the arteries supplying skeletal muscle than on those supplying skin. It is used in the treatment of peripheral vascular disease and in premature labor.


Raynaud disease

An episodic vasoconstriction resulting in discoloration of the skin and pain in the affected areas, often involving fingers or toes. Classically associated with triphasic color changes (white, blue, red) but may be biphasic. Often occurs in response to cold temperatures or emotional stress. May be primary or secondary to an underlying autoimmune disease.


dyspepsia

An uncomfortable, often painful feeling in the stomach, resulting from impaired digestion. Symptoms include burning stomach pain, bloating, heartburn, nausea, and vomiting. Causes include gastritis, gastric ulcer, gastroesophageal reflux disease, pancreatic disease, and gallbladder disease.


Nonproductive cough

A cough that does not produce phlegm or mucus.


Aminoglutethimide

An aromatase inhibitor that is used in the treatment of advanced BREAST CANCER.

A dicarboximide that is a six-membered cyclic compound having ethyl and 4-aminophenyl substituents at the 3-position.


Secondary malignant neoplasm of female breast


Flibanserin

a serotonin receptor modulator with high affinity for the following serotonin (5-hydroxytryptamine or 5-T) receptors: agonist activity at 5-HT1A and antagonist activity at 5-HT2A. Flibanserin also has moderate antagonist activities at the 5-HT2B, 5-HT2C, and dopamine D4 receptors, used in the treatment of premenopausal women with hypoactive sexual desire disorder

An N-alkylpiperazine that is 1-[2-(1,3-dihydro-2-oxobenzimidazol-1-yl)ethyl]piperazine in which the remaining amino proton is replaced by a 3-(trifluoromethyl)phenyl group. A multifunctional serotonin agonist and antagonist which is used for the treatment of pre-menopausal women with hypoactive sexual desire disorder.


hypoactive sexual desire disorder

A disorder characterized by a recurrent or persistent lack of desire for sexual activity. The lack of sexual desire is not attributable to another psychiatric disorder or to the physiological effects of substance use or a general medical condition.


Decreased liver function

Reduced ability of the liver to perform its functions.


DIBOTERMIN ALFA

a member of the TGF-beta superfamily and closely related to the osteogenic/bone morphogenetic proteins


intervertebral disk degenerative disorder

Any disease of a degenerative nature that affects the intervertebral disk.


Fractured tibia

A partial or complete breakage of the tibia.


SARGRAMOSTIM

a glycosylated form of recombinant GM-CSF; used to treat drug-induced agranulocytosis; amino acid sequence known


Oprelvekin is a recombinant human Interleukin-11. Interleukin eleven (IL-11) is a thrombopoietic growth factor that directly stimulates the proliferation of hematopoietic stem cells and megakaryocyte progenitor cells and induces megakaryocyte maturation resulting in increased platelet production.


EVOLOCUMAB

Evolocumab is a human monoclonal IgG2 directed against human proprotein convertase subtilisin kexin 9 (PCSK9). Evolocumab binds to PCSK9 and inhibits circulating PCSK9 from binding to the low density lipoprotein (LDL) receptor (LDLR), preventing PCSK9-mediated LDLR degradation and permitting LDLR to recycle back to the liver cell surface. By inhibiting the binding of PCSK9 to LDLR, evolocumab increases the number of LDLRs available to clear LDL from the blood, thereby lowering LDL-C levels.


Familial hypercholesterolemia - heterozygous


Befunolol

synonym BFE 60 refers to HCl; RN given refers to parent cpd; structure


Rolapitant

An orally bioavailable, centrally-acting, selective, neurokinin 1 receptor (NK1-receptor) antagonist with potential antiemetic activity. Upon oral administration, rolapitant competitively binds to and blocks the activity of the NK1-receptor in the central nervous system, thereby inhibiting the binding of the endogenous ligand, substance P (SP). This may prevent both SP-induced emesis and chemotherapy-induced nausea and vomiting (CINV). The interaction of SP with the NK1-receptor plays a key role in the induction of nausea and vomiting caused by emetogenic cancer chemotherapy. Compared to other NK1-receptor antagonists, rolapitant has both a more rapid onset of action and a much longer half-life.

An azaspiro compound that is 1,7-diazaspiro[4.5]decan-2-one carrying additional phenyl and 1-{[3,5-bis(trifluoromethyl)phenyl]ethoxy}methyl substituents at position 8. Used (in the form of the hydrochloride hydrate) for the prevention of delayed nausea and vomiting associated with initial and repeat courses of emetogenic cancer chemotherapy.


schizophrenia

A major psychotic disorder characterized by abnormalities in the perception or expression of reality. It affects the cognitive and psychomotor functions. Common clinical signs and symptoms include delusions, hallucinations, disorganized thinking, and retreat from reality.


Flupenthixol

A thioxanthene neuroleptic that, unlike CHLORPROMAZINE, is claimed to have CNS-activating properties. It is used in the treatment of psychoses although not in excited or manic patients. (From Martindale, The Extra Pharmacopoeia, 30th ed, p595)


Domperidone

A specific blocker of dopamine receptors. It speeds gastrointestinal peristalsis, causes prolactin release, and is used as antiemetic and tool in the study of dopaminergic mechanisms.

1-[3-(Piperidin-1-yl)propyl]-1,3-dihydro-2H-benzimidazol-2-one in which the 4-position of the piperidine ring is substituted by a 5-chloro-1,3-dihydro-2H-benzimidazol-2-on-1-yl group. A dopamine antagonist, it is used as an antiemetic for the short-term treatment of nausea and vomiting, and to control gastrointestinal effects of dopaminergic drugs given in the management of parkinsonism. The free base is used in oral suspensions, while the maleate salt is used in tablet preparations.


Nausea and vomiting

Nausea is a commonly encountered symptom that has been defined as an unpleasant painless subjective feeling that one will imminently vomit. Vomiting has been defined as the forceful expulsion of the contents of the stomach, duodenum, or jejunum through the oral cavity. While nausea and vomiting are often thought to exist on a temporal continuum, this is not always the case. There are situations when severe nausea may be present without emesis and less frequently, when emesis may be present without preceding nausea.


Carfenazine

A member of the class of phenothiazines that is 10H-phenothiazine which carries a propanoyl group at position 2 and a 3-[4-(2-hydroxyethyl)piperazin-1-yl]propyl group at position N-10. It is an antipsychotic drug that is used for the treatment of chronic schizophrenia.


Mesoridazine

A phenothiazine antipsychotic with effects similar to CHLORPROMAZINE.

A phenothiazine substituted at position 2 (para to the S atom) by a methylsulfinyl group, and on the nitrogen by a 2-(1-methylpiperidin-2-yl)ethyl group.


Fluspirilene

A long-acting injectable antipsychotic agent used for chronic schizophrenia.


ALIROCUMAB

antibody to PCSK9 to manage LDL cholesterol


Hypercholesterolemia

An increased concentration of cholesterol in the blood.


syndromic dyslipidemia

A inherited lipid metabolism disorder that is part of a larger syndrome.


atherosclerosis

Build-up of fatty material and calcium deposition in the arterial wall resulting in partial or complete occlusion of the arterial lumen.


ANAKINRA

A ligand that binds to but fails to activate the INTERLEUKIN 1 RECEPTOR. It plays an inhibitory role in the regulation of INFLAMMATION and FEVER. Several isoforms of the protein exist due to multiple ALTERNATIVE SPLICING of its mRNA. As IL-1 levels are elevated in patients with COVID-19, anakinra is currently being investigated for its potential to combat cytokine release syndrome (CRS) in patients with severely ill COVID-19.


cryopyrin-associated periodic syndrome

Cryopyrin associated periodic syndrome (CAPS) defines a group of autoinflammatory diseases, characterized by recurrent episodes of systemic inflammatory attacks in the absence of infection or autoimmune disease. CAPS comprises 3 disorders on a continuum of severity: severe CINCA syndrome, intermediate Muckle-Wells syndrome (MWS) and milder familial cold urticaria (FCAS).


CINCA syndrome

Chronic Infantile Neurological, Cutaneous, and Articular (CINCA) syndrome is characterized by skin rash, joint involvement, chronic meningitis with granulocytes and, in some cases, sensorineural hearing loss and ocular signs.


Eliglustat tartrate

recombinant protein for treatment of Fabry disease

A tartrate that is the hemitartrate salt of eliglustat. A ceramide glucosyltransferase inhibitor used (as its tartrate salt) for treatment of Gaucher's disease.


Fabry disease

Fabry disease (FD) is a progressive, inherited, multisystemic lysosomal storage disease characterized by specific neurological, cutaneous, renal, cardiovascular, cochleo-vestibular and cerebrovascular manifestations.


TUROCTOCOG ALFA

a recombinant coagulation factor VIII for prevention and treatment of bleeding in haemophilia A


Gadoversetamide

nonionic, linear chelate used for magnetic resonance imaging of brain and spine

A gadolinium coordination entity that consists of Gd(3+) coordinated to 3,6,9-triazaundecadiamide in which each of the amide nitrogens is substituted by a 2-methoxyethyl group and in which the nitrogens at positions 3, 6, and 9 are each substituted by carboxylatomethyl group. The gadolinium is coordinated to the three tertiary amino groups as well as to the carboxylate groups. A white odourless powder that is freely soluble in water, gadoversetamide has paramagnetic properties and is used as a contrast agent in magnetic resonance imaging. It distributes mainly in extracellular fluid, but does not cross the blood-brain barrier. It is used particularly in imaging the brain, spine and liver.


AGALSIDASE ALFA

genetically engineered, glycosylated human alpha-galactosidase isoenzyme A used in treatment of Fabry disease


Sacubitril

inhibits neprilysin (neutral endopeptidase; NEP) via LBQ657, the active metabolite and leads to increased levels of peptides that are degraded by neprilysin such as natriuretic peptides. It has been approved in combination with valsartan (under the name ENTRESTO) for the treatment of heart failure.


Trelagliptin

Trelagliptin is an orally active dipeptidyl peptidase (DPP)-4 inhibitor developed by Takeda and approved in Japan for the treatment of type 2 diabetes mellitus.


Pirenzepine

An antimuscarinic agent that inhibits gastric secretion at lower doses than are required to affect gastrointestinal motility, salivary, central nervous system, cardiovascular, ocular, and urinary function. It promotes the healing of duodenal ulcers and due to its cytoprotective action is beneficial in the prevention of duodenal ulcer recurrence. It also potentiates the effect of other antiulcer agents such as CIMETIDINE and RANITIDINE. It is generally well tolerated by patients.


Neridronic acid

used for therapy of Paget's disease of bone & malignant hypercalcaemia


osteoporosis

A condition of reduced bone mass, with decreased cortical thickness and a decrease in the number and size of the trabeculae of cancellous bone (but normal chemical composition), resulting in increased fracture incidence. Osteoporosis is classified as primary (Type 1, postmenopausal osteoporosis; Type 2, age-associated osteoporosis; and idiopathic, which can affect juveniles, premenopausal women, and middle-aged men) and secondary osteoporosis (which results from an identifiable cause of bone mass loss).


Incadronic acid

increases serum 1,25-dihydroxyvitamin D in rats via stimulating renal 1-hydroxylase activity; structure given in first source


humoral hypercalcemia of malignancy

Hypercalcemia generally develops as a late complication of malignancy; its appearance has grave prognostic significance. It remains unclear, however, whether death is associated with hypercalcemic crisis (uncontrolled or recurrent progressive hypercalcemia) or with advanced disease. Symptoms include central nervous system impairment such as delirium with prominent symptoms of personality change, cognitive dysfunction, disorientation, incoherent speech, and psychotic symptoms such as hallucinations and delusions, smooth muscle hypotonicity, and altered cardiovascular function.


Brexpiprazole

an atypical antipsychotic the efficacy of brexpiprazole may be mediated through a combination of partial agonist activity at serotonin 5-HT1A and dopamine D2 receptors, and antagonist activity at serotonin 5-HT2A receptors


major depressive disorder

An episode of depression lasting two or more weeks without an intervening episode of mania.


PEGVISOMANT

is a genetically engineered analogue of human growth hormone that functions as a growth hormone-receptor antagonist; used to treat Acromegaly


HYOSCYAMINE

An atropine with a 2S-configuration.


VELAGLUCERASE ALFA

produced by gene activation technology in a human fibroblast cell line and has the same amino acid sequence as the naturally occurring human enzyme glucocerebrosidase


Orphenadrine

A muscarinic antagonist used to treat drug-induced parkinsonism and to relieve pain from muscle spasm.

A tertiary amino compound which is the phenyl-o-tolylmethyl ether of 2-(dimethylamino)ethanol.


parkinsonian disorder

A group of disorders which feature impaired motor control characterized by bradykinesia, MUSCLE RIGIDITY; TREMOR; and postural instability. Parkinsonian diseases are generally divided into primary parkinsonism (see PARKINSON DISEASE), secondary parkinsonism (see PARKINSON DISEASE, SECONDARY) and inherited forms. These conditions are associated with dysfunction of dopaminergic or closely related motor integration neuronal pathways in the BASAL GANGLIA.


Trastuzumab emtansine

Immunotoxin that consists of humanized monoclonal anti-HER2 antibody TRASTUZUMAB covalently linked to anti-microtubule agent MAYTANSINOID DM1 for treatment of metastatic breast cancer in patients who previously received trastuzumab and a TAXANES, separately or in combination.


HER2 positive breast carcinoma

A biologic subset of breast carcinoma defined by high expression of HER2, GRB7, and TRAP100, and by lack of expression of estrogen receptor (ER).


Lipegfilgrastim

A long acting glyco-pegylated recombinant form of human granulocyte colony-stimulating factor (G-CSF), with hematopoietic activity. Similar to G-CSF, lipegfilgrastim binds to and activates specific cell surface receptors, and stimulates neutrophil progenitor proliferation and differentiation. Therefore, this agent may prevent the duration and incidence of chemotherapy-induced neutropenia. Compared to filgrastim, lipegfilgrastim has a prolonged plasma half-life.


Moxifloxacin

A fluoroquinolone that acts as an inhibitor of DNA TOPOISOMERASE II and is used as a broad-spectrum antibacterial agent.

A quinolone that consists of 4-oxo-1,4-dihydroquinoline-3-carboxylic acid bearing a cyclopropyl substituent at position 1, a fluoro substitiuent at position 6, a (4aS,7aS)-octahydro-6H-pyrrolo[3,4-b]pyridin-6-yl group at position 7 and a methoxy substituent at position 8. A member of the fluoroquinolone class of antibacterial agents.


pneumonic plague

A plague in which the bacteria have infected the lungs.


septicemic plague

A plague in which the bacteria have entered the bloodstream.


RASBURICASE

a recombinant form of enzyme that degrades uric acid to allantoin just downstream of xanthine oxidase; used to treat hyperuricaemia


Mixture of antifungal heptaene macrolides from Streptomyces griseus or Actinomyces levoris used topically in candidiasis. The antibiotic complex is composed of candicidins A, B, C, and D, of which D is the major component.


Somatropin recombinant

A 191-amino acid polypeptide hormone secreted by the human adenohypophysis (PITUITARY GLAND, ANTERIOR), also known as GH or somatotropin. Synthetic growth hormone, termed somatropin, has replaced the natural form in therapeutic usage such as treatment of dwarfism in children with growth hormone deficiency.


Prader-Willi syndrome

Prader-Willi syndrome is a rare genetic disorder characterized by hypothalamic-pituitary abnormalities with severe hypotonia during the neonatal period and first two years of life and the onset of hyperphagia with a risk of morbid obesity during infancy and adulthood, learning difficulties and behavioral problems or severe psychiatric problems.


pituitary dwarfism

Proportionately decreased bodily growth due to failure of the pituitary gland to produce an adequate supply of growth hormone.


Turner syndrome

Turner syndrome is a chromosomal disorder associated with the complete or partial absence of an X chromosome.


Epoetinum alfa

Glycoprotein hormone, secreted chiefly by the KIDNEY in the adult and the LIVER in the FETUS, that acts on erythroid stem cells of the BONE MARROW to stimulate proliferation and differentiation.


ROMIPLOSTIM

Romiplostim, a member of the TPO mimetic class, is an Fc-peptide fusion protein (peptibody) that activates intracellular transcriptional pathways leading to increased platelet production via the TPO receptor (also known as cMpl). The peptibody molecule contains two identical single-chain subunits, each consisting of human immunoglobulin IgG1 Fc domain, covalently linked at the C-terminus to a peptide containing two thrombopoietin receptor-binding domains. Romiplostim has no amino acid sequence homology to endogenous TPO. Romiplostim is produced by recombinant DNA technology in Escherichia coli.


autoimmune thrombocytopenia

An autoimmune form of thrombocytopenia.


LARONIDASE

Laronidase is a polymorphic variant of the human enzyme alpha‑l‑iduronidase that is produced by recombinant DNA technology which catalyzes the hydrolysis of terminal alpha-l-iduronic acid residues of dermatan sulfate and heparan sulfate. Reduced or absent alpha-L-iduronidase activity results in the accumulation of the glycosaminoglycans (GAG) substrates, dermatan sulfate and heparan sulfate, throughout the body and leads to widespread cellular, tissue, and organ dysfunction.


mucopolysaccharidosis type 1

The most common type of mucopolysaccharidosis. It is inherited in an autosomal recessive pattern. It comprises a group of lysosomal storage diseases which includes the most severe form (Hurler syndrome) and the mildest form (Scheie syndrome).


Aluminum Acetate

astringent for temporary relief of minor skin irritations


Skin Irritation

A mild inflammatory dermal tissue reaction; it can be caused by physical contact with an irritant or can be a local response to a systemic trigger.


ELOSULFASE ALFA

BMN-100 and BMN 110 are recombinant GALNS; RefSeq NM_000512


mucopolysaccharidosis type 4A

A rare autosomal recessive lysosomal storage disease caused by deficiency of the enzyme galactosamine-6-sulfatase. It is characterized by skeletal and central nervous system deficits.


Camostat

A benzoate ester resulting from the formal condensation of the carboxy group of 4-guanidinobenzoic acid with the hydroxy group of 2-(dimethylamino)-2-oxoethyl (4-hydroxyphenyl)acetate. It is a potent inhibitor of the human transmembrane protease serine 2 (TMPRSS2) and its mesylate salt is currently under investigation for its effectiveness in COVID-19 patients.


Gastro-esophageal reflux disease with esophagitis


Xipamide

A sulfamoylbenzamide analog of CLOPAMIDE. It is diuretic and saluretic with antihypertensive activity. It is bound to PLASMA PROTEINS, thus has a delayed onset and prolonged action.


Edema

An abnormal accumulation of fluid beneath the skin, or in one or more cavities of the body.


Lumiracoxib

A selective non-steroidal anti-inflammatory drug (NSAID) that belongs to the group cyclo-oxygenase 2 (COX-2) inhibitors. It blocks the COX-2 enzyme, resulting in a reduction in the production of prostaglandins, substances that are involved in the inflammation process.

An amino acid that is phenylacetic acid which is substituted at position 2 by the nitrogen of 2-chloro-6-fluoroaniline and at position 5 by a methyl group. A highly selective cyclooxygenase 2 inhibitor, it was briefly used for the treatment of osteoarthritis, but was withdrawn due to concersns of hepatotoxicity.


Azapropazone

An anti-inflammatory agent used in the treatment of rheumatoid arthritis. It also has uricosuric properties and has been used to treat gout.

A member of the class of benzotriazines that is 1,2-dihydro-1,2,4-benzotriazine bearing a dimethylamino substitutent at position 3 and a methyl substituent at position 7 and in which the nitrogens at positions 1 and 2 are both acylated by a carboxy group of propylmalonic acid.


ankylosing spondylitis

An autoimmune chronic inflammatory disorder characterized by inflammation in the vertebral joints of the spine and sacroiliac joints. It predominantly affects young males. Patients present with stiffness and pain in the spine.


PEGFILGRASTIM

pegylated (r-G-CSF) conjugated to polyethylene glycol for treatment of neutropenic patients with non-myeloid malignancies who are at high risk for infection


FILGRASTIM

A recombinant granulocyte colony-stimulating factor (G-CSF) that is used in the treatment and prevention of NEUTROPENIA, preparation and collection of blood progenitor cells, and for use in PERIPHERAL BLOOD STEM CELL TRANSPLANTATION.


severe congenital neutropenia

A congenital disorder characterized by chronic neutropenia with absolute neutrophil count less than 500/uL and arrest of neutrophil maturation at the promyelocyte/myelocyte level. It is inherited in autosomal recessive or autosomal dominant patterns. Sporadic cases have also been reported. It is usually diagnosed in the first year of life. Patients present with frequent and life-threatening infections.


cyclic hematopoiesis

A hematologic disorder caused by a mutation in the ELANE (ELA2) gene; clinical manifestations include recurrent neutropenia with resultant susceptibility to infection leading to fever.


Chronic idiopathic neutropenia

A rare hematologic disorder characterized by neutropenia lasting more than three months with no identifiable cause.


Flufenamic acid

An anthranilic acid derivative with analgesic, anti-inflammatory, and antipyretic properties. It is used in musculoskeletal and joint disorders and administered by mouth and topically. (From Martindale, The Extra Pharmacopoeia, 30th ed, p16)

An aromatic amino acid consisting of anthranilic acid carrying an N-(trifluoromethyl)phenyl substituent. An analgesic and anti-inflammatory, it is used in rheumatic disorders.


Poldine

major descriptor (66-86); on-line search BENZILATES (66-86); INDEX MEDICUS search POLDINE (66-86); RN given refers to parent cpd


Ivabradine

Ivabradine is a pure heart rate lowering agent, acting by selective and specific inhibition of the cardiac pacemaker If current that controls the spontaneous diastolic depolarisation in the sinus node and regulates heart rate. The cardiac effects are specific to the sinus node with no effect on intra-atrial, atrioventricular or intraventricular conduction times, nor on myocardial contractility or ventricular repolarisation.

A member of the class of benzazepines that is 7,8-dimethoxy-1,3,4,5-tetrahydro-3-benzazepin-2-one in which the amide hydrogen is replaced by a [{[(7S)-3,4-dimethoxybicyclo[4.2.0]octa-1,3,5-trien-7-yl]methyl}(methyl)amino]propyl} group. Used (as its hydrochloride salt) to treat patients with angina who have intolerance to beta blockers and/or heart failure.


Triamcinolone hexacetonide

synthetic glucocorticoid analog used for  anti-inflammatory effects in disorders of many organ systems


diffuse alopecia areata

An alopecia areata that involves diffuse loss of hair over the whole scalp.


discoid lupus erythematosus

A chronic, autoimmune skin condition that manifests with a red, scaling rash, most often found on the face, ears, and scalp; these lesions often lead to permanent scarring and dyspigmentation. Patients may have lesions with or without other symptoms or antibodies suggestive of systemic lupus erythematosus (SLE).


keloid

An irregularly shaped, elevated mark on the skin caused by deposits of excessive amounts of collagen during wound healing. It extends beyond the original boundaries of the wound and may enlarge progressively.


lichen planus

A chronic, recurrent, pruritic inflammatory disorder of unknown etiology that affects the skin and mucus membranes. It presents with rashes and papules that tend to resolve spontaneously. It may be associated with hepatitis C. Certain drugs that contain arsenic or bismuth are associated with reactions mimicking lichen planus.


neurodermatitis

Skin findings arising from repeated rubbing, picking or scratching of a real or imagined irritation of the skin.


SILTUXIMAB

a chimeric anti-interleukin-6 antibody for treatment of multiple myeloma and renal cell carcinoma; in phase I and phase II clinical trials. It was investigated for its potential to combat cytokine release syndrome (CRS) in patients with severely ill COVID-19 but the efficacy and safety of siltuximab in the treatment of COVID-19 were not established.


Castleman disease

Castleman disease (CD) is a benign lymphoproliferative disorder that may present as a localized or multicentric form. The clinical manifestations are heterogeneous, ranging from asymptomatic discrete lymphadenopathy to recurrent episodes of diffuse lymphadenopathy with severe systemic symptoms.


Amfetaminil

a supposedly insoluble amphetamine complex that may release amphetamine; proposed as central nervous system stimulant; minor desriptor (78-86); on line & INDEX MEDICUS search AMPHETAMINES (75-86); RN given refers to parent compound


ETANERCEPT

A recombinant version of soluble human TNF receptor fused to an IgG FC fragment that binds specifically to TUMOR NECROSIS FACTOR and inhibits its binding with endogenous TNF receptors. It prevents the inflammatory effect of TNF and is used to treat RHEUMATOID ARTHRITIS; PSORIATIC ARTHRITIS and ANKYLOSING SPONDYLITIS.


psoriatic arthritis

Joint inflammation associated with psoriasis.


Sepsis

Systemic inflammatory response to infection.


Roflumilast

A benzamide obtained by formal condensation of the carboxy group of 3-(cyclopropylmethoxy)-4-(difluoromethoxy)benzoic acid with the amino group of 3,5-dichloropyridin-4-amine. Used for treatment of bronchial asthma and chronic obstructive pulmonary disease.


Severe chronic obstructive pulmonary disease


TRASTUZUMAB

The HER2 (or c-erbB2) proto-oncogene encodes a transmembrane receptor protein of 185 kDa, which is structurally related to the epidermal growth factor receptor. Trastuzumab products have been shown, in both in vitro assays and in animals, to inhibit the proliferation of human tumor cells that overexpress HER2. Trastuzumab products are mediators of antibody-dependent cellular cytotoxicity (ADCC). In vitro, trastuzumab product-mediated ADCC has been shown to be preferentially exerted on HER2 overexpressing cancer cells compared with cancer cells that do not overexpress HER2.


Palbociclib

Palbociclib is an inhibitor of cyclin-dependent kinases (CDK) 4 and 6. Cyclin D1 and CDK4/6 are downstream of signaling pathways which lead to cellular proliferation. In vitro, palbociclib reduced cellular proliferation of estrogen receptor (ER)-positive breast cancer cell lines by blocking progression of the cell from G1 into S phase of the cell cycle. Treatment of breast cancer cell lines with the combination of palbociclib and antiestrogens leads to decreased retinoblastoma (Rb) protein phosphorylation resulting in reduced E2F expression and signaling, and increased growth arrest compared to treatment with each drug alone. In vitro treatment of ER-positive breast cancer cell lines with the combination of palbociclib and antiestrogens led to increased cell senescence compared to each drug alone, which was sustained for up to 6 days following palbociclib removal and was greater if antiestrogen treatment was continued. In vivo studies using a patient-derived ER-positive breast cancer xenograft model demonstrated that the combination of palbociclib and letrozole increased the inhibition of Rb phosphorylation, downstream signaling, and tumor growth compared to each drug alone.

A member of the class of pyridopyrimidines that is 2-{[5-(piperazin-1-yl)pyridin-2-yl]amino}pyrido[2,3-d]pyrimidin-7-one bearing additional methyl, acetyl and cyclopentyl substituents at positions 5, 6 and 8 respectively. It is used in combination with letrozole for the treatment of metastatic breast cancer.


Her2-receptor negative breast cancer

A breast adenocarcinoma which is negative for the expression of the HER2 receptor.


thyroid gland follicular carcinoma

A differentiated adenocarcinoma arising from the follicular cells of the thyroid gland. The nuclear features which characterize the thyroid gland papillary carcinoma are absent. Radiation exposure is a risk factor and it comprises approximately 10% to 15% of thyroid cancers. Clinically, it usually presents as a solitary mass in the thyroid gland. It is generally unifocal and thickly encapsulated and shows invasion of the capsule or the vessels. Diagnostic procedures include thyroid ultrasound and fine needle biopsy.


thyroid gland papillary carcinoma

A differentiated adenocarcinoma arising from the follicular cells of the thyroid gland. Radiation exposure is a risk factor and it is the most common malignant thyroid lesion, comprising 75% to 80% of all thyroid cancers in iodine sufficient countries. Diagnostic procedures include thyroid ultrasound and fine needle biopsy. Microscopically, the diagnosis is based on the distinct characteristics of the malignant cells, which include enlargement, oval shape, elongation, and overlapping of the nuclei. The nuclei also display clearing or have a ground glass appearance.


Arformoterol

the (R,R)-enantiomer of formoterol, is a selective long-acting beta2-adrenergic receptor agonist (beta2-agonist) that has two-fold greater potency than racemic formoterol

An N-[2-hydroxy-5-(1-hydroxy-2-{[1-(4-methoxyphenyl)propan-2-yl]amino}ethyl)phenyl]formamide in which both of the stereocentres have R configuration. The active enantiomer of formoterol, it is administered by inhalation (generally as the tartrate salt) as a direct-acting sympathomimetic and  bronchodilator for the treatment of chronic obstructive pulmonary disease (any progressive respiratory disease that makes it harder to breathe over time, such as chronic bronchitis and emphysema).


chronic obstructive pulmonary disease

A chronic and progressive lung disorder characterized by the loss of elasticity of the bronchial tree and the air sacs, destruction of the air sacs wall, thickening of the bronchial wall, and mucous accumulation in the bronchial tree. The pathologic changes result in the disruption of the air flow in the bronchial airways. Signs and symptoms include shortness of breath, wheezing, productive cough, and chest tightness. The two main types of chronic obstructive pulmonary disease are chronic obstructive bronchitis and emphysema.


pulmonary emphysema

A subcategory of chronic obstructive pulmonary disease (COPD). It occurs in people who smoke and suffer from chronic bronchitis. It is characterized by inflation of the alveoli, alveolar wall damage, and reduction in the number of alveoli, resulting in difficulty breathing.


Apremilast

Apremilast is an oral small-molecule inhibitor of phosphodiesterase 4 (PDE4) specific for cyclic adenosine monophosphate (cAMP). PDE4 inhibition results in increased intracellular cAMP levels. The specific mechanism(s) by which apremilast exerts its therapeutic action in psoriatic arthritis patients and psoriasis patients is not well defined.

A member of the class of isoindoles that is isoindole-1,3-dione substituted at position 4 by an acetamido group and at position 1 by a 1-(3-ethoxy-4-methoxyphenyl)-2-(methylsulfonyl)ethyl group. Used for treatment of psoriatic arthritis.


Tenofovir alafenamide

a phosphonoamidate prodrug of tenofovir (2'deoxyadenosine monophosphate analog). Plasma exposure to tenofovir alafenamide allows for permeation into cells and then tenofovir alafenamide is intracellularly converted to tenofovir through hydrolysis by cathepsin A. Tenofovir is subsequently phosphorylated by cellular kinases to the active metabolite tenofovir diphosphate. Tenofovir diphosphate inhibits HIV replication through incorporation into viral DNA by the HIV reverse transcriptase, which results in DNA chain-termination

An L-alanine derivative that is isopropyl L-alaninate in which one of the amino hydrogens is replaced by an (S)-({[(2R)-1-(6-amino-9H-purin-9-yl)propan-2-yl]oxy}methyl)(phenoxy)phosphoryl group. A prodrug for tenofovir, it is used (as the fumarate salt) in combination therapy for the treatment of HIV-1 infection.


Epalrestat

A monocarboxylic acid that is 1,3-thiazolidine which is substituted on the nitrogen by a carboxymethyl group, at positions 2 and 4 by thioxo and oxo groups, respectively, and at position 5 by a 2-methyl-3-phenylprop-2-en-1-ylidene group. It is an inhibitor of aldose reductase (which catalyses the conversion of glucose to sorbitol) and is used for the treatment of some diabetic complications, including neuropathy.


diabetic neuropathy

A chronic, pathological complication associated with diabetes mellitus, where nerve damages are incurred due to diabetic microvascular injury involving small blood vessels that supply these nerves, resulting in peripheral and/or autonomic nerve dysfunction.


Panobinostat

An indole and hydroxamic acid derivative that acts as a HISTONE DEACETYLASE inhibitor. It is used as an antineoplastic agent in combination with BORTEZOMIB and DEXAMETHASONE for the treatment of MULTIPLE MYELOMA.

A hydroxamic acid obtained by formal condensation of the carboxy group of (2E)-3-[4-({[2-(2-methylindol-3-yl)ethyl]amino}methyl)phenyl]prop-2-enoic acid with the amino group of hydroxylamine. A histone deacetylase inhibitor used (as its lactate salt) in combination with bortezomib and dexamethasone for the treatment of multiple myeloma.


Avibactam

a non-beta-lactam beta-lactamase inhibitor that inactivates some beta-lactamases and protects ceftazidime from degradation by certain beta-lactamases

A member of the class of azabicycloalkanes that is (2S,5R)-7-oxo-1,6-diazabicyclo[3.2.1]octane-2-carboxamide in which the amino hydrogen at position 6 is replaced by a sulfooxy group. Used (in the form of its sodium salt) in combination with ceftazidime pentahydrate for the treatment of complicated urinary tract infections including pyelonephritis.


pyelonephritis

An inflammatory process affecting the kidney. The cause is most often bacterial, but may also be fungal in nature. Signs and symptoms may include fever, chills, flank pain, painful and frequent urination, cloudy or bloody urine, and confusion.


Abdominal Infection

Infection in the abdominal cavity resulting from injury, acute intestinal inflammation (e.g., acute appendicitis), intestinal perforation, or complication of abdominal surgery.


Febuxostat

A thiazole derivative and inhibitor of XANTHINE OXIDASE that is used for the treatment of HYPERURICEMIA in patients with chronic GOUT.

A 1,3-thiazolemonocarboxylic acid that is 4-methyl-1,3-thiazole-5-carboxylic acid which is substituted by a 3-cyano-4-(2-methylpropoxy)phenyl group at position 2. It is an orally-active, potent, and selective xanthine oxidase inhibitor used for the treatment of chronic hyperuricaemia in patients with gout.


Tolvaptan

A benzazepine derivative and selective VASOPRESSIN V2 RECEPTOR antagonist that is used to treat euvolemic and hypervolemic HYPONATREMIA. It is also used in the treatment of rapidly progressing AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE to slow the rate of cyst development and renal insufficiency.

A benzazepine derivative incorporating a benzenedicarboxamide function whch is a selective vasopressin V2 receptor antagonist used to treat euvolemic and hypervolemic hyponatremia. It is also used in the treatment of rapidly progressing autosomal dominant polycystic kidney disease to slow the rate of cyst development and renal insufficiency. Tolvaptan is a racemate consisting of a 1:1 mixture of 5R and 5S stereomers.


autosomal dominant polycystic kidney disease

Autosomal dominant form of polycystic kidney disease.


squamous cell lung carcinoma

A carcinoma arising from squamous bronchial epithelial cells. It may be keratinizing or non-keratinizing. Keratinizing squamous cell carcinoma is characterized by the presence of keratinization, pearl formation, and/or intercellular bridges. Non-keratinizing squamous cell carcinoma is characterized by the absence of keratinization, pearl formation, and intercellular bridges. Cigarette smoking and arsenic exposure are strongly associated with squamous cell lung carcinoma.


trichomoniasis

An infection that is caused by Trichomonas.


Aminolevulinic acid

A compound produced from succinyl-CoA and GLYCINE as an intermediate in heme synthesis. It is used as a PHOTOCHEMOTHERAPY for actinic KERATOSIS.

The simplest delta-amino acid in which the hydrogens at the gamma position are replaced by an oxo group. It is metabolised to protoporphyrin IX, a photoactive compound which accumulates in the skin. Used (in the form of the hydrochloride salt)in combination with blue light illumination for the treatment of minimally to moderately thick actinic keratosis of the face or scalp.


Roxatidine acetate

a histamine H2 receptor antagonist drug used to treat gastric ulcers


gastroesophageal reflux disease

A chronic disorder characterized by reflux of the gastric and/or duodenal contents into the distal esophagus. It is usually caused by incompetence of the lower esophageal sphincter. Symptoms include heartburn and acid indigestion. It may cause injury to the esophageal mucosa.


Zollinger-Ellison syndrome

Zollinger-Ellison syndrome (ZES) is characterized by severe peptic disease (ulcers/esophageal disease) caused by hypergastrinemia secondary to a gastrinoma resulting in increased gastric acid secretion.


IDURSULFASE

An enzyme that specifically cleaves the ester sulfate of iduronic acid. Its deficiency has been demonstrated in Hunter's syndrome, which is characterized by an excess of dermatan sulfate and heparan sulfate. EC 3.1.6.13.


mucopolysaccharidosis type 2

A lysosomal storage disease leading to a massive accumulation of glycosaminoglycans and a wide variety of symptoms including distinctive coarse facial features, short stature, cardio-respiratory involvement and skeletal abnormalities. It manifests as a continuum varying from a severe to an attenuated form without neuronal involvement.


Benzydamine

A benzyl-indazole having analgesic, antipyretic, and anti-inflammatory effects. It is used to reduce post-surgical and post-traumatic pain and edema and to promote healing. It is also used topically in treatment of RHEUMATIC DISEASES and INFLAMMATION of the mouth and throat.

A member of the class of indazoles carrying benzyl and 3-(dimethylamino)propyl groups at positions 1 and 3 respectively. A locally-acting nonsteroidal anti-inflammatory drug that also exhibits local anaesthetic and analgesic properties.


atopic conjunctivitis

Conjunctivitis due to hypersensitivity to various allergens.


Finafloxacin

Finafloxacin belongs to the fluoroquinolone class of antibacterials which involves the inhibition of bacterial type II topoisomerase enzymes, DNA gyrase and topoisomerase IV, which are required for bacterial DNA replication, transcription, repair and recombination.

A quinolone that is 4-oxo-1,4-dihydroquinoline-3-carboxylic acid which is substituted at positions 1, 6, 7 and 8 by cyclopropyl, fluoro, hexahydropyrrolo[3,4-b][1,4]oxazin-6-yl and cyano groups respectively; an antibiotic used for treatment of acute otitis externa (swimmer's ear) caused by the bacteria Pseudomonas aeruginosa and Staphylococcus aureus.


acute otitis externa

Acute form of otitis externa.


Beraprost

stable prostacyclin analog; structure given in first source

An organic heterotricyclic compound that is (3aS,8bS)-2,3,3a,8b-tetrahydro-1H-benzo[b]cyclopenta[d]furan in which the hydrogens at positions 1R, 2R and 5 are replaced by (3S)-3-hydroxy-4-methyloct-1-en-6-yn-1-yl, hydroxy and 3-carboxypropyl groups, respectively. It is a prostaglandin receptor agonist which is approved to treat pulmonary arterial hypertension in Asia.


pulmonary arterial hypertension

Pulmonary arterial hypertension (PAH) is a group of diseases characterized by mean pulmonary artery pressure >20 mmHg and elevated pulmonary arterial resistance leading to right heart failure. PAH is progressive and potentially fatal. PAH may be idiopathic and/ or familial, have overt features of venous/capillary involvement (pulmonary veno-occlusive disease, PVOD/pulmonary capillary hemangiomatosis, PCH), induced by drug or toxin (drug-or toxin-induced PAH), or associated with other diseases like congenital heart disease, connective tissue disease, HIV, schistosomiasis, portal hypertension (PAH associated with other disease).


Safinamide

Safinamide acts through both dopaminergic and non-dopaminergic mechanisms of action. Safinamide is a highly selective and reversible MAO-B inhibitor causing an increase in extracellular levels of dopamine in the striatum. Safinamide is associated with state-dependent inhibition of voltage-gated sodium (Na+) channels, and modulation of stimulated release of glutamate. To what extent the nondopaminergic effects contribute to the overall effect has not been established.


Ceftolozane belongs to the cephalosporin class of antibacterial drugs. The bactericidal action of ceftolozane results from inhibition of cell wall biosynthesis, and is mediated through binding to penicillin-binding proteins (PBPs). Ceftolozane is an inhibitor of PBPs of P. aeruginosa (e.g., PBP1b, PBP1c, and PBP3) and E. coli (e.g., PBP3).


Fenticonazole

A racemate comprising equimolar amounts of (R)- and (S)-fenticonazole. Used (as its nitrate salt) for the treatment of vaginal candidiasis.


vulvovaginal candidiasis

Infection of the vulva and vagina with a fungus of the genus CANDIDA. It is a disease associated with HIV infection.


Liraglutide

An analog of GLUCAGON-LIKE PEPTIDE 1 and agonist of the GLUCAGON-LIKE PEPTIDE 1 RECEPTOR that is used as a HYPOGLYCEMIC AGENT and supplemental therapy in the treatment of DIABETES MELLITUS by patients who do not respond to METFORMIN.

A lipopeptide that is an analogue of human GLP-1 in which the lysine residue at position 27 is replaced by arginine and a hexadecanoyl group attached to the remaining lysine via a glutamic acid spacer. Used as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus.


Tibolone

used in prevention of postmenopausal osteoporosis

Estran-3-one with a double bond between positions 5 and 10, and bearing both an ethynyl group and a hydroxy group at position 17 (R-configuration). A synthetic steroid hormone drug which acts as an agonist at all five type I steroid hormone receptors, it is used in the prevention of postmenopausal osteoporosis and for treatment of endometriosis.


postmenopausal osteoporosis

Metabolic disorder associated with fractures of the femoral neck, vertebrae, and distal forearm. It occurs commonly in women within 15-20 years after menopause, and is caused by factors associated with menopause including estrogen deficiency.


Rimonabant

A pyrazole and piperidine derivative that acts as a selective cannabinoid type-1 receptor (CB1 RECEPTOR) antagonist. It inhibits the proliferation and maturation of ADIPOCYTES, improves lipid and glucose metabolism, and regulates food intake and energy balance. It is used in the management of OBESITY.

A carbohydrazide obtained by formal condensation of the carboxy group of 5-(4-chlorophenyl)-1-(2,4-dichlorophenyl)-4-methyl-1H-pyrazole-3-carboxylic acid with the amino group of 1-aminopiperidine. It is a potent and selective cannabinoid receptor 1 (CB1R) antagonist. Besides its antagonistic properties, numerous studies have shown that, at micromolar concentrations rimonabant behaves as an inverse agonist at CB1 receptors. The drug was the first selective CB1 antagonist/inverse agonist introduced into clinical practice to treat obesity and metabolic-related disorders. It was later withdrawn from market due to CNS-related adverse effects including depression and suicidal ideation.


Reboxetine

A morpholine derivative that is a selective and potent noradrenaline reuptake inhibitor; it is used in the treatment of DEPRESSIVE DISORDER.


Bezafibrate

An antilipemic agent that lowers CHOLESTEROL and TRIGLYCERIDES. It decreases LOW DENSITY LIPOPROTEINS and increases HIGH DENSITY LIPOPROTEINS.

A monocarboxylic acid amide obtained by the formal condensation of the carboxy group of 4-chlorobenzoic acid with the amino group of 2-[4-(2-aminoethyl)phenoxy]-2-methylpropanoic acid. Benafibrate is used for the treatment of hyperlipidaemia.


Tetracosactide

A synthetic peptide that is identical to the 24-amino acid segment at the N-terminal of ADRENOCORTICOTROPIC HORMONE. ACTH (1-24), a segment similar in all species, contains the biological activity that stimulates production of CORTICOSTEROIDS in the ADRENAL CORTEX.

A synthetic peptide that is identical to the 24-amino acid segment at the N-terminal of adrenocorticotropic hormone (corticotropin). A segment similar in all species, it contains the biological activity that stimulates production of corticosteroids in the adrenal cortex. It is used diagnostically to investigate adrenocortical insufficiency.


Tienilic acid

a novel diuretic with uricosuric action, it has been used as an antihypertensive agent, withdrawn shortly after approval due to liver toxicity

An aromatic ketone that is 2,3-dichlorophenoxyacetic acid in which the hydrogen at position 4 on the benzene ring is replaced by a thiophenecarbonyl group. A loop diuretic used to treat hypertension, it was withdrawn from the market in 1982 due to links with hepatitis.


Lutein

A xanthophyll found in the major LIGHT-HARVESTING PROTEIN COMPLEXES of plants. Dietary lutein accumulates in the MACULA LUTEA.


age-related macular degeneration

Age-related loss of vision in the central portion of the retina (macula), secondary to retinal degeneration.


Posterior blepharitis

A type of blepharitis that affects the meibomian glands and meibomian gland orifices. This abnormality can be associated with a spectrum of appearances ranging from meibomian seborrhoea (foaming meibomian gland secretions) and meibomianitis (inflamed meibomian glands), to chalazia.


BLINATUMOMAB

a single-chain bispecific recombinant antibody from Micromet's BiTE (bispecific T-cell engager) product platform that binds both the CD19 antigen and the T-cell receptor (CD3), for the potential treatment of B-cell lymphoma


B Acute Lymphoblastic Leukemia

A type of ALL characterized by elevated levels of B-cell lymphoblasts in the bone marrow and the blood.


acquired polycythemia vera

Polycythemia vera (PV) is an acquired myeloproliferative disorder characterized by an elevated absolute red blood cell mass caused by uncontrolled red blood cell production, frequently associated with uncontrolled white blood cell and platelet production.


Lidocaine

A local anesthetic and cardiac depressant used as an antiarrhythmia agent. Its actions are more intense and its effects more prolonged than those of PROCAINE but its duration of action is shorter than that of BUPIVACAINE or PRILOCAINE.

The monocarboxylic acid amide resulting from the formal condensation of N,N-diethylglycine with 2,6-dimethylaniline.


premature ejaculation

A disorder characterized by persistent or recurrent ejaculation before or after penetration and before the person wishes it.


Prilocaine

A local anesthetic that is similar pharmacologically to LIDOCAINE. Currently, it is used most often for infiltration anesthesia in dentistry.

An  amino acid amide in which N-propyl-DL-alanine and 2-methylaniline have combined to form the amide bond; used as a local anaesthetic.


AFLIBERCEPT

has antiangiogenic activity; consists of the second and third Ig domains of VEGFR1 and VEGFR2, respectively, fusion to the Fc region of IgG1; putative antineoplastic agent that binds all VEGF-A isoforms; possible medicine for eye disorders


wet macular degeneration

A form of RETINAL degeneration in which abnormal CHOROIDAL NEOVASCULARIZATION occurs under the RETINA and MACULA LUTEA, causing bleeding and leaking of fluid. This leads to bulging and or lifting of the macula and the distortion or destruction of central vision.


central retinal vein occlusion with macular edema


Branch retinal vein occlusion with macular edema


metastasis from malignant tumor of colon


Netupitant

Netupitant is a selective antagonist of human substance P/neurokinin 1 (NK-1) receptors. Delayed emesis has been largely associated with the activation of tachykinin family neurokinin 1 (NK-1) receptors (broadly distributed in the central and peripheral nervous systems) by substance P. As shown in in vitro and in vivo studies, netupitant inhibits substance P mediated responses.

A monocarboxylic acid amide obtained by formal condensation of the carboxy group of 2-[3,5-bis(trifluoromethyl)phenyl]-2-methylpropanoic acid with the secondary amino group of N-methyl-4-(2-methylphenyl)-6-(4-methylpiperazin-1-yl)pyridin-3-amine; an antiemetic used in combination with palonosetron hydrochloride (under the trade name Akynzeo) to treat nausea and vomiting in patients undergoing cancer chemotherapy.


Ledipasvir

an inhibitor of the HCV NS5A protein, which is required for viral replication

A benzimidazole derivative that is used in combination with sofosbuvir (under the trade name Harvoni) for the treatment of chronic hepatitis C genotype 1 infection.


gastric ulcer

An ulcerated lesion in the mucosal surface of the stomach. It may progress to involve the deeper layers of the gastric wall.


Sepsis caused by Streptococcus pneumoniae


Diquafosol

A pyrimidine ribonucleoside 5'-tetraphosphate compound having 5'-uridinyl residues at the P(1)- and P(4)-positions.


Lantus Solostar

A recombinant LONG ACTING INSULIN and HYPOGLYCEMIC AGENT that is used to manage BLOOD GLUCOSE in patients with DIABETES MELLITUS.


type 1 diabetes mellitus

A chronic condition characterized by minimal or absent production of insulin by the pancreas.


Lafutidine

histamine H(2)-receptor antagonist used as an antiulcer drug


Duloxetine

A thiophene derivative and selective NEUROTRANSMITTER UPTAKE INHIBITOR for SEROTONIN and NORADRENALINE (SNRI). It is an ANTIDEPRESSIVE AGENT and ANXIOLYTIC, and is also used for the treatment of pain in patients with DIABETES MELLITUS and FIBROMYALGIA.


female stress incontinence

The involuntary loss of urine in females secondary to insufficient strength of the pelvic floor muscles; this can result from physical changes following pregnancy and childbirth, or as a response to a decrease in estrogen during menopause.


NINTEDANIB

A member of the class of oxindoles that is a kinase inhibitor used (in the form of its ethylsulfonate salt) for the treatment of idiopathic pulmonary fibrosis and cancer.


interstitial lung disease 2

A nonneoplastic pulmonary disease that is characterized by the formation of scar tissue within the lungs in the absence of any known cause.


Crohn disease

A gastrointestinal disorder characterized by chronic inflammation involving all layers of the intestinal wall, noncaseating granulomas affecting the intestinal wall and regional lymph nodes, and transmural fibrosis. Crohn disease most commonly involves the terminal ileum; the colon is the second most common site of involvement.


ulcerative colitis

An inflammatory bowel disease involving the mucosal surface of the large intestine and rectum. It may present with an acute or slow onset and follows an intermittent or continuous course. Signs and symptoms include abdominal pain, diarrhea, fever, weight loss, and intestinal hemorrhage.


Benznidazole

Benznidazole inhibits the synthesis of DNA, RNA, and proteins within the T. cruzi parasite. Studies suggest that benznidazole is reduced by a Type I nitroreductase (NTR) enzyme of T. cruzi producing a series of short-lived intermediates that may promote damage to several macromolecules including DNA. In mammalian cells, however, benznidazole is metabolized by reduction of the nitro group to an amino group by a Type II NTR enzyme. The precise mechanism of action is not known. Benznidazole is active against all three stages, trypomastigotes, amastigotes, and epimastigotes, of T. cruzi. However, the sensitivity of T. cruzi strains to benznidazole, from different geographic regions, may vary.

A monocarboxylic acid amide obtained by formal condensation of the carboxy group of (2-nitroimidazol-1-yl)acetic acid with the aromatic amino group of benzylamine. Used for treatment of Chagas disease.


Chagas disease

A parasitic infection caused by Trypanosoma cruzi. It is transmitted by insect bites. It is characterized by an acute and chronic phase; in the acute phase patients may have fever, malaise, and swelling at the site of the insect bite. In the chronic phase patients develop hepatosplenomegaly, lymphadenopathy, cardiomyopathy and arrhythmias.


Diethylcarbamazine

An anthelmintic used primarily as the citrate in the treatment of filariasis, particularly infestations with Wucheria bancrofti or Loa loa.


elephantiasis

Enlargement of an area of the body due to obstruction within the lymphatic system and the resulting accumulation of lymph.


Eflornithine

An inhibitor of ORNITHINE DECARBOXYLASE, the rate limiting enzyme of the polyamine biosynthetic pathway.

A fluoroamino acid that is ornithine substituted by a difluoromethyl group at position 2.


human African trypanosomiasis

A parasitic disorder caused by protozoa of the Trypanosoma brucei species. It is transmitted by flies and is endemic in various regions of Sub-Saharan Africa. Signs and symptoms include fever, joint pain, headache, and significant swelling of the lymph nodes. If left untreated, the parasitic infection causes anemia, heart, kidney, and endocrine failure, and neurologic damage. Subsequently patients develop confusion, disruption of the sleep cycle, and mental deterioration. The infection may lead to coma and death.


Melarsoprol

Arsenical used in trypanosomiases. It may cause fatal encephalopathy and other undesirable side effects.


Nifurtimox

A nitrofuran thiazine that has been used against TRYPANOSOMIASIS.


Pentostam

Antimony complex where the metal may exist in either the pentavalent or trivalent states. The pentavalent gluconate is used in leishmaniasis. The trivalent gluconate is most frequently used in schistosomiasis.


leishmaniasis

Infectious disease that is transmitted through the bite of hematophagous female phlebotomine sand flies. The clinical spectrum ranges from asymptomatic to clinically overt disease which can remain localized to the skin or disseminate to the upper oral and respiratory mucous membranes or throughout the reticulo-endothelial system. Three main clinical syndromes have been described: visceral (or Kala-Azar; with fever, weight loss, hepatosplenomegaly), cutaneous, and mucocutaneous leishmaniasis (cutaneous or mucocutaneous ulceration).


Suramin

A polyanionic compound with an unknown mechanism of action. It is used parenterally in the treatment of African trypanosomiasis and it has been used clinically with diethylcarbamazine to kill the adult Onchocerca. (From AMA Drug Evaluations Annual, 1992, p1643) It has also been shown to have potent antineoplastic properties.

A member of the class of phenylureas that is urea in which each of the amino groups has been substituted by a 3-({2-methyl-5-[(4,6,8-trisulfo-1-naphthyl)carbamoyl]phenyl}carbamoyl)phenyl group. An activator of both the rabbit skeletal muscle RyR1 and sheep cardiac RyR2 isoform ryanodine receptor channels, it has been used for the treatment of human African trypanosomiasis for over 100 years.


Pentamidine

Antiprotozoal agent effective in trypanosomiasis, leishmaniasis, and some fungal infections; used in treatment of PNEUMOCYSTIS pneumonia in HIV-infected patients. It may cause diabetes mellitus, central nervous system damage, and other toxic effects.

A diether consisting of pentane-1,5-diol in which both hydroxyl hydrogens have been replaced by 4-amidinophenyl groups. A  trypanocidal drug that is used for treatment of cutaneous leishmaniasis and Chagas disease.


DULAGLUTIDE

a glucagon-like peptide-1 receptor agonist; GLP-1 immunoglobulin G (IgG4) Fc fusion protein with extended activity


Beclomethasone dipropionate

An anti-inflammatory, synthetic glucocorticoid. It is used topically as an anti-inflammatory agent and in aerosol form for the treatment of ASTHMA.

A steroid ester comprising beclomethasone having propionyl groups at the 17- and 21-positions.


vasomotor rhinitis

Inflammation in the nasal cavity mucosa that results from the abnormal regulation of the blood flow in the nose. It may be caused by temperature fluctuations, air pollution, strong odors, and tobacco smoke. It results in chronic nasal congestion, sneezing, and running nose.


essential hypertension

Hypertension that presents without an identifiable cause.


Cilazapril

One of the ANGIOTENSIN-CONVERTING ENZYME INHIBITORS (ACE inhibitors) used for hypertension. It is a prodrug that is hydrolyzed after absorption to its main metabolite cilazaprilat.

A pyridazinodiazepine resulting from the formal condensation of the carboxy group of cilazaprilat with ethanol. It is a drug used in the treatment of hypertension and heart failure.


Imidapril

A member of the class of imidazolidines that is (4S)-1-methyl-2-oxoimidazolidine-4-carboxylic acid in which the hydrogen of the imidazolidine nitrogen has been substituted by (1S)-1-{[(2S)-1-ethoxy-1-oxo-4-phenylbutan-2-yl]amino}ethyl group. It is the prodrug for imidaprilat, an ACE inhibitor used for the treatment of chronic heart failure.


Cetilistat

lipase inhibitor in randomized, placebo-controlled study of weight reduction in obese patients (3/2007)


Trichlormethiazide

A thiazide diuretic with properties similar to those of HYDROCHLOROTHIAZIDE. (From Martindale, The Extra Pharmacopoeia, 30th ed, p830)

A benzothiadiazine, hydrogenated at positions 2, 3 and 4 and substituted with an aminosulfonyl group at C-7, a chloro substituent at C-6 and a dichloromethyl group at C-3 and with S-1 as an S,S-dioxide. A sulfonamide antibiotic, it is used as a diuretic to treat oedema (including that associated with heart failure) and hypertension.


influenza

An acute viral infection of the respiratory tract, occurring in isolated cases, in epidemics, or in pandemics; it is caused by serologically different strains of viruses (influenzaviruses) designated A, B, and C, has a 3-day incubation period, and usually lasts for 3 to 10 days. It is marked by inflammation of the nasal mucosa, pharynx, and conjunctiva; headache; myalgia; often fever, chills, and prostration; and occasionally involvement of the myocardium or central nervous system.


CALFACTANT

Infasurf is tradename; a lung surfactant extract from new born calves containing 35 mg/mL phospholipid & 2% surfactant-associated proteins B & C (wt/wt)


Cobicistat

A carbamate and thiazole derivative that functions as a CYTOCHROME P450 CYP3A INHIBITOR to enhance the concentration of ANTI-HIV AGENTS, with which it is used in combination, for the treatment of HIV INFECTIONS.

A monocarboxylic acid amide obtained by formal condensation of the carboxy group of (2S)-2-({[(2-isopropyl-1,3-thiazol-4-yl)methyl](methyl)carbamoyl}amino)-4-(morpholin-4-yl)butanoic acid with the amino group of 1,3-thiazol-5-ylmethyl [(2R,5R)-5-amino-1,6-diphenylhexan-2-yl]carbamate. Acts as a pharmacoenhancer in treatment of HIV-1 by inhibiting P450 enzymes that metabolise other medications..


head and neck neoplasm

A benign or malignant neoplasm that affects the anatomic structures of the head and neck region. Representative examples of benign neoplasms include salivary gland pleomorphic adenoma and nasal cavity papilloma. Representative examples of malignant neoplasms include oral cavity squamous cell carcinoma, laryngeal squamous cell carcinoma, and salivary gland carcinoma.


squamous cell carcinoma

A carcinoma arising from squamous epithelial cells. Morphologically, it is characterized by the proliferation of atypical, often pleomorphic squamous cells. Squamous cell carcinomas are graded by the degree of cellular differentiation as well, moderately, or poorly differentiated. Well differentiated carcinomas are usually associated with keratin production and the presence of intercellular bridges between adjacent cells. Representative examples are lung squamous cell carcinoma, skin squamous cell carcinoma, and cervical squamous cell carcinoma.


Clivarin

Low-molecular-weight fragment of heparin, having a 4-enopyranosuronate sodium structure at the non-reducing end of the chain. It is prepared by depolymerization of the benzylic ester of porcine mucosal heparin. Therapeutically, it is used as an antithrombotic agent. (From Merck Index, 11th ed)


Deep venous thrombosis

Formation of a blot clot in a deep vein. The clot often blocks blood flow, causing swelling and pain. The deep veins of the leg are most often affected.


CID 3081884

A random polymer of L-ALANINE, L-GLUTAMIC ACID, L-LYSINE, and L-TYROSINE that structurally resembles MYELIN BASIC PROTEIN. It is used in the treatment of RELAPSING-REMITTING MULTIPLE SCLEROSIS.


multiple sclerosis

A progressive autoimmune disorder affecting the central nervous system resulting in demyelination. Patients develop physical and cognitive impairments that correspond with the affected nerve fibers.


Oritavancin

A semisynthetic glycopeptide used (as its bisphosphate salt) for the treatment of acute bacterial skin and skin structure infections caused or suspected to be caused by susceptible isolates of designated Gram-positive microorganisms including MRSA.


Enviomycin

Cyclic basic peptide related to VIOMYCIN. It is isolated from an induced mutant of Streptomyces griseoverticillatus var. tuberacticus and acts as an antitubercular agent with less ototoxicity than tuberactinomycin.


pulmonary tuberculosis

A bacterial infection that affects the lungs and is caused by Mycobacterium tuberculosis. Most patients with tuberculosis do not have symptoms (latent tuberculosis) and are not contagious. When signs and symptoms occur (active tuberculosis), patients become contagious. The signs and symptoms include chronic cough with blood-tinged sputum, night sweats, fever, fatigue, and weight loss.


Suvorexant

a highly selective antagonist for orexin receptors OX1R and OX2R, the orexin neuropeptide signaling system is a central promoter of wakefulness, blocking the binding of wake-promoting neuropeptides orexin A and orexin B to receptors OX1R and OX2R is thought to suppress wake drive

An aromatic amide obtained by formal condensation of the carboxy group of 5-methyl-2-(2H-1,2,3-triazol-2-yl)benzoic acid with the secondary amino group of 5-chloro-2-[(5R)-5-methyl-1,4-diazepan-1-yl]-1,3-benzoxazole. An orexin receptor antagonist used for the management of insomnia.


insomnia

A sleep disorder characterized by difficulty in falling asleep and/or remaining asleep.


Dexetimide

A muscarinic antagonist that has been used to treat neuroleptic-induced parkinsonism. Benzetimide is the (-)-enantimorph of dexetimide.


Eliglustat

Eliglustat tartrate is a small molecule inhibitor of glucosylceramide synthase that resembles the ceramide substrate for the enzyme. Gaucher disease is caused by a deficiency of the lysosomal enzyme acid beta-glucosidase. Acid beta-glucosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide. The enzymatic deficiency causes an accumulation of glucosylceramide (GL-1) primarily in the lysosomal compartment of macrophages, giving rise to foam cells or "Gaucher cells".

A carboxamide obtained by formal condensation of the carboxy group of octanoic acid with the primary amino group of (1R,2R)-2-amino-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-3-(pyrrolidin-1-yl)propan-1-ol. A ceramide glucosyltransferase inhibitor used (as its tartrate salt) for treatment of Gaucher's disease.


Gaucher disease

Gaucher disease (GD) is a lysosomal storage disorder encompassing three main forms (types 1, 2 and 3), a fetal form and a variant with cardiac involvement (Gaucher disease - ophthalmoplegia - cardiovascular calcification or Gaucher-like disease).


chronic gastritis

Inflammation of the stomach that is chronic in nature.


Coenzyme Q10

A ubiquinone having a side chain of 10 isoprenoid units. In the naturally occurring isomer, all isoprenyl double bonds are in the E- configuration.


Istradefylline

selective antagonist at the A2A receptor, used in the treatment of Parkinson's disease


hyperlipidemia

Elevated levels of lipids in the blood.


hypotensive disorder

Blood pressure that is abnormally low.


Efinaconazole

Efinaconazole is an azole antifungal. Efinaconazole inhibits fungal lanosterol 14α-demethylase involved in the biosynthesis of ergosterol, a constituent of fungal cell membranes.

A member of the class of triazoles that is butan-2-ol which is substituted at positions 1, 2, and 3 by 1,2,4-triazol-1-yl, 2,4-difluorophenyl, and 4-methylenepiperidin-1-yl groups, respectively (the 2R,3R stereoisomer). It is an antifungal drug used for the topical treatment of onychomycosis (a nail infection caused mainly by dermatophytes).


tinea unguium

A fungal infection of the nail, usually caused by dermatophytes; yeasts; or nondermatophyte molds.


Onychomycosis due to Trichophyton mentagrophytes


Miltefosine

hexadecyl phosphocholine derivative of cisplatin; did not substantially activate HIV long terminal repeat; less toxic than cisplatin

A phospholipid that is the hexadecyl monoester of phosphocholine.


Pregnancy

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Sjogren-Larsson syndrome

A neurocutaneous disorder caused by an inborn error of lipid metabolism and characterized by congenital ichthyosis, intellectual deficit, and spasticity.


Daclatasvir

A member of the class of biphenyls that is a potent inhibitor of nonstructural protein 5A and is used (as its hydrochloride salt) for treatment of hepatitis C.


a hydroxamate-type inhibitor of histone deacetylase


Ibandronic Acid

Aminobisphosphonate that is a potent inhibitor of BONE RESORPTION. It is used in the treatment of HYPERCALCEMIA associated with malignancy, for the prevention of fracture and bone complications in patients with breast cancer and bone metastases, and for the treatment and prevention of POSTMENOPAUSAL OSTEOPOROSIS.


hypercalcemia disease

Abnormally high concentration of calcium in the peripheral blood.