Erdheim-Chester disease

Erdheim-Chester disease (ECD), a non-Langerhans form of histiocytosis, is a multisystemic disease characterized by various manifestations such as skeletal involvement with bone pain, exophthalmos, diabetes insipidus, renal impairment and central nervous system (CNS) and/or cardiovascular involvement.


Chronic cough

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A persistent cough, defined as a cough lasting longer than eight weeks in adults or longer than four weeks in children.


chronic hiccup

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Chronic hiccup is a rare movement disorder characterized by involuntary spasmodic contractions of the inspiratory muscles synchronized with larynx closure lasting for more than 48 hours.


Intestinal hemorrhage due to angiodysplasia of intestine


Gastric hemorrhage due to angiodysplasia of stomach


traumatic encephalopathy

Encephalopathy resulting from trauma.


Cannabis hyperemesis syndrome co-occurrent and due to cannabis abuse


Cannabis hyperemesis syndrome co-occurrent and due to cannabis dependence


hypokalemic periodic paralysis

Hypokalemic periodic paralysis (hypoPP) is characterized by episodes of muscle paralysis lasting from a few to 24-48 hours and associated with a fall in blood potassium levels.


Overdose of beta-adrenergic blocking drug


Nerve injury

Injury to nervous tissue.


mucositis

Inflammation of the mucous membranes.


wound care

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viral myocarditis

Myocarditis that is caused by an infection with a viral agent.


Mastalgia

Pain in the breast.


complex regional pain syndrome

Complex regional pain syndrome (CRPS) is a rare neurologic disease painful progressive condition that corresponds to a group of disorders characterized by a disproportionate spontaneous or stimulus-induced pain, accompanied by a variably mixed myriad of autonomic and motor disorders including symptoms such as swelling, allodynia, skin blood supply and trophic disturbances. CRPS most often affects one of the arms, legs, hands, or feet and usually occurs after an injury or trauma to that limb.


Sexual dysfunction due to substance

A disorder characterized by clinically significant sexual dysfunction (including impaired desire, impaired arousal, impaired orgasm, or sexual pain) that is fully explained by substance use.


Night sweats

Occurrence of excessive sweating during sleep.


Neuropathic Pain

Chronic pain caused by damage to nerve fibers.  It is usually associated with tissue injury.


Nightmares associated with chronic post-traumatic stress disorder


AMIVANTAMAB

Amivantamab-vmjw is a bispecific antibody that binds to the extracellular domains of EGFR and MET. In in vitro and in vivo studies amivantamab-vmjw was able to disrupt EGFR and MET signaling functions through blocking ligand binding and, in exon 20 insertion mutation models, degradation of EGFR and MET. The presence of EGFR and MET on the surface of tumor cells also allows for targeting of these cells for destruction by immune effector cells, such as natural killer cells and macrophages, through antibody-dependent cellular cytotoxicity ( ADCC) and trogocytosis mechanisms, respectively.


Non-small cell lung cancer with mutation in epidermal growth factor receptor


ADUCANUMAB

Aducanumab-avwa is a human, immunoglobulin gamma 1 (IgG1) monoclonal antibody directed against aggregated soluble and insoluble forms of amyloid beta. The accumulation of amyloid beta plaques in the brain is a defining pathophysiological feature of Alzheimer’s disease. ADUHELM reduces amyloid beta plaques, as evaluated in Studies 1, 2, and 3.


Finerenone

Finerenone is a nonsteroidal, selective antagonist of the mineralocorticoid receptor (MR), which is activated by aldosterone and cortisol and regulates gene transcription. Finerenone blocks MR mediated sodium reabsorption and MR overactivation in both epithelial (e.g., kidney) and nonepithelial (e.g., heart, and blood vessels) tissues. MR overactivation is thought to contribute to fibrosis and inflammation. Finerenone has a high potency and selectivity for the MR and has no relevant affinity for androgen, progesterone, estrogen, and glucocorticoid receptors.


Chronic kidney disease due to type 2 diabetes mellitus


Fexinidazole

Studies with Trypanosoma brucei and other protozoans suggest that, like for other nitrocontaining drugs, the nitroreductase (NTR) enzyme plays an important role in the bioactivation of fexinidazole resulting in generation of reactive amines and damage to DNA and proteins. The activity of fexinidazole and its metabolites (M1 and M2) is trypanocidal and appears to be concentration and time dependent. However, the precise mechanism by which fexinidazole and the two metabolites exhibit activity against T. brucei is not known.


Belumosudil is an inhibitor of rho-associated, coiled-coil containing protein kinase (ROCK) which inhibits ROCK2 and ROCK1 with IC50 values of approximately 100 nanoM and 3 microM, respectively. Belumosudil downregulated proinflammatory responses via regulation of STAT3/STAT5 phosphorylation and shifting Th17/Treg balance in ex-vivo or in vitro-human T cell assays. Belumosudil also inhibited aberrant pro-fibrotic signaling, in vitro. In vivo, belumosudil demonstrated activity in animal models of chronic GVHD.


Odevixibat

Odevixibat is a reversible inhibitor of the ileal bile acid transporter (IBAT). It decreases the reabsorption of bile acids (primarily the salt forms) from the terminal ileum. Pruritus is a common symptom in patients with PFIC and the pathophysiology of pruritus in patients with PFIC is not completely understood. Although the complete mechanism by which odevixibat improves pruritus in PFIC patients is unknown, it may involve inhibition of the IBAT, which results in decreased reuptake of bile salts, as observed by a decrease in serum bile acids.


progressive familial intrahepatic cholestasis

Progressive familial intrahepatic cholestasis (PFIC) refers to a heterogeneous group of autosomal recessive disorders of childhood that disrupt bile formation and present with cholestasis of hepatocellular origin.


Belzutifan

Belzutifan is an inhibitor of hypoxia-inducible factor 2 alpha (HIF-2alpha), with potential antineoplastic activity. HIF-2alpha is a transcription factor that plays a role in oxygen sensing by regulating genes that promote adaptation to hypoxia. Under normal oxygen levels, HIF-2alpha is targeted for ubiquitin-proteasomal degradation by VHL protein. Lack of functional VHL protein results in stabilization and accumulation of HIF-2alpha. Upon stabilization, HIF-2alpha translocates into the nucleus and interacts with hypoxia-inducible factor 1 beta (HIF-1beta) to form a transcriptional complex that induces expression of downstream genes, including genes associated with cellular proliferation, angiogenesis, and tumor growth. Belzutifan binds to HIF-2alpha, and in conditions of hypoxia or impairment of VHL protein function, belzutifan blocks the HIF-2alpha-HIF-1beta interaction, leading to reduced transcription and expression of HIF-2alpha target genes. In vivo, belzutifan demonstrated anti-tumor activity in mouse xenograft models of renal cell carcinoma.


von Hippel-Lindau disease

A familial cancer predisposition syndrome associated with a variety of malignant and benign neoplasms, most frequently retinal, cerebellar, and spinal hemangioblastoma, renal cell carcinoma (RCC), and pheochromocytoma.


Difelikefalin

Difelikefalin is a kappa opioid receptor (KOR) agonist. The relevance of KOR activation to therapeutic effectiveness is not known.


Mobocertinib

Mobocertinib is a kinase inhibitor of the epidermal growth factor receptor (EGFR) that irreversibly binds to and inhibits EGFR exon 20 insertion mutations at lower concentrations than wild type (WT) EGFR. Two pharmacologically-active metabolites (AP32960 and AP32914) with similar inhibitory profiles to mobocertinib have been identified in the plasma after oral administration of mobocertinib. In vitro, mobocertinib also inhibited the activity of other EGFR family members (HER2 and HER4) and one additional kinase (BLK) at clinically relevant concentrations (IC50 values <2 nM).


Atogepant

Atogepant is a calcitonin gene-related peptide (CGRP) receptor antagonist.

A secondary carboxamide resulting from the formal condensation of the carboxy group of (3'S)-2'-oxo-1',2',5,7-tetrahydrospiro[cyclopenta[b]pyridine-6,3'-pyrrolo[2,3-b]pyridine]-3-carboxylic acid with the amino group of (3S,5S,6R)-3-amino-6-methyl-1-(2,2,2-trifluoroethyl)-5-(2,3,6-trifluorophenyl)piperidin-2-one. It is a selective oral, small-molecule antagonist of calcitonin gene-related peptide (CGRP) receptor that has been approved for the treatment of migraine.


ANIFROLUMAB

Anifrolumab-fnia is a human IgG1kappa monoclonal antibody that binds to subunit 1 of the type I interferon receptor (IFNAR) with high specificity and affinity. This binding inhibits type I IFN signaling, thereby blocking the biologic activity of type I IFNs. Anifrolumab-fnia also induces the internalization of IFNAR1, thereby reducing the levels of cell surface IFNAR1 available for receptor assembly. Blockade of receptor mediated type I IFN signaling inhibits IFN responsive gene expression as well as downstream inflammatory and immunological processes. Inhibition of type I IFN blocks plasma cell differentiation and normalizes peripheral T-cell subsets. Type I IFNs play a role in the pathogenesis of SLE. Approximately 60-80% of adult patients with active SLE express elevated levels of type I IFN inducible genes.


AVALGLUCOSIDASE ALFA

Avalglucosidase alfa-ngpt provides an exogenous source of GAA. The M6P on avalglucosidase alfa-ngpt mediates binding to M6P receptors on the cell surface with high affinity. After binding, it is internalized and transported into lysosomes where it undergoes proteolytic cleavage that results in increased GAA enzymatic activity. Avalglucosidase alfa-ngpt then exerts enzymatic activity in cleaving glycogen.


glycogen storage disease due to acid maltase deficiency, late-onset

Glycogen storage disease due to acid maltase deficiency, late onset (AMDL), a form of Glycogen storage disease due to acid maltase deficiency (AMD), a degenerative metabolic myopathy particularly affecting respiratory and skeletal muscles, is characterized by an accumulation of glycogen in lysosomes.


LONAPEGSOMATROPIN

Lonapegsomatropin is a pegylated human growth hormone (somatropin) for once-weekly subcutaneous injection. Somatropin binds to the growth hormone (GH) receptor in the cell membrane of target cells resulting in intracellular signal transduction and a host of pharmacodynamic effects. Somatropin has direct tissue and metabolic effects, and indirect effects mediated by insulin-like growth factor-1 (IGF-1), including stimulation of chondrocyte differentiation and proliferation, stimulation of hepatic glucose output, protein synthesis and lipolysis. Somatropin stimulates skeletal growth in pediatric patients with growth hormone deficiency (GHD) as a result of effects on the growth plates (epiphyses) of long bones.


Somatotropin deficiency

Abnormally low levels of circulating somatotropin.


TISOTUMAB VEDOTIN

Tisotumab vedotin-tftv is a tissue factor (TF)-directed antibody drug conjugate (ADC). The antibody is a human IgG1 directed against cell surface TF. TF is the primary initiator of the extrinsic blood coagulation cascade. The small molecule, MMAE, is a microtubule-disrupting agent, attached to the antibody via a protease-cleavable linker. Nonclinical data suggests that the anticancer activity of tisotumab vedotin-tftv is due to the binding of the ADC to TF expressing cancer cells, followed by internalization of the ADC-TF complex, and release of MMAE via proteolytic cleavage. MMAE disrupts the microtubule network of actively dividing cells, leading to cell cycle arrest and apoptotic cell death. In vitro, tisotumab vedotin-tftv also mediates antibody-dependent cellular phagocytosis and antibody-dependent cellular cytotoxicity.


BIMEKIZUMAB

Bimekizumab is a humanised IgG1/kappa monoclonal antibody that selectively binds with high affinity to IL-17A, IL-17F and IL-17AF cytokines, blocking their interaction with the IL-17RA/IL-17RC receptor complex. Elevated concentrations of IL-17A and IL-17F have been implicated in the pathogenesis of several immune-mediated inflammatory diseases including plaque psoriasis. Bimekizumab inhibits these proinflammatory cytokines, resulting in the normalization of skin inflammation and as a consequence improvement in clinical symptoms associated with psoriasis. From in vitro models, bimekizumab was shown to inhibit psoriasis-related gene expression and cytokine production to a greater extent than inhibition of IL-17A alone.


obesity due to pro-opiomelanocortin deficiency

Pro-opiomelanocortin (POMC) deficiency is a form of monogenic obesity resulting in severe early-onset obesity, adrenal insufficiency, red hair and pale skin.


Obesity due to leptin receptor gene deficiency


TRALOKINUMAB

Tralokinumab is a fully human IgG4 monoclonal antibody that specifically binds to the type 2 cytokine interleukin-13 (IL-13) and inhibits its interaction with the IL-13 receptors. Tralokinumab neutralises the biological activity of IL-13 by blocking its interaction with the IL-13Ralpha1/IL-4Ralpha receptor complex. IL-13 is a major driver of human type 2 inflammatory disease, such as atopic dermatitis and inhibiting the IL-13 pathway with tralokinumab in patients decreases many of the mediators of type 2 inflammation.


Vosoritide

Vosoritide is a modified type C natriuretic peptide (CNP). In patients with achondroplasia, endochondral bone growth is negatively regulated due to a gain of function mutation in fibroblast growth factor receptor 3 (FGFR3). Binding of vosoritide to natriuretic peptide receptor-B (NPR-B) antagonises FGFR3 downstream signalling by inhibiting the extracellular signal-regulated kinases 1 and 2 (ERK1/2) in the mitogen-activated protein kinase (MAPK) pathway at the level of rapidly accelerating fibrosarcoma serine/threonine protein kinase (RAF-1). As a result, vosoritide, like CNP, acts as a positive regulator of endochondral bone growth as it promotes chondrocyte proliferation and differentiation.


Achondroplasia

Achondroplasia is the most common form of chondrodysplasia, characterized by rhizomelia, exaggerated lumbar lordosis, brachydactyly, and macrocephaly with frontal bossing and midface hypoplasia.


pemphigus vulgaris

Pemphigus is a group of chronic autoimmune skin diseases characterized by blister formations on the outer layer of the skin and the mucous membranes. Three clinical forms have been characterized, of which pemphigus vulgaris is the most frequent (75%).


Ioflupane I-123

Ioflupane binds reversibly to the human recombinant dopamine transporter (DaT). Following administration of DaTscan to humans, radioactive decay of the iodine 123 emits gamma radiation which can be detected externally using gamma detectors, allowing visualization of the brain striata through SPECT imaging. Autoradiography of post-mortem human brain slices exposed to radiolabeled ioflupane shows concentration of the radiolabel in striatum (caudate nucleus and putamen). The specificity of the binding of ioflupane I 125 to dopamine transporter was demonstrated by competition studies with the DaT inhibitor GBR 12909 (a dopamine reuptake inhibitor), the serotonin reuptake inhibitor citalopram, and the norepinephrine reuptake inhibitor desipramine in post-mortem human brain slices exposed to radiolabeled ioflupane.  Citalopram reduced binding in the neocortex and thalamus with only minor effects in the striatum. This indicated that the binding in the cortex and thalamus is mainly to the serotonin reuptake sites.  Desipramine showed no effect on the level of striatal binding of ioflupane I 125, but reduced extrastriatal binding by 60 to 85%.  The binding of ioflupane I 125 to the striatum was abolished in the presence of high concentrations of GBR 12909, indicating selectivity of ioflupane binding for the pre-synaptic DaT.

An azabicycloalkane that is ecgonine methyl ester in which the N-methyl group is replaced by 3-fluoropropyl and the 3beta-hydroxy group is replaced by 4-((123)I)iodophenyl. Used for the imaging of dopamine transporters in the brain of adult patients with potential Parkinsonian Syndromes.


Behcet disease

A chronic, relapsing, multisystemic vasculitis characterized by mucocutaneous lesions, as well as articular, vascular, ocular and central nervous system manifestations.


PEFICITINIB

Peficitinib inhibits the signal transduction of cytokines important for the pathological condition of RA as it inhibits the kinase activity of the JAK family (JAK1, JAK2, JAK3, and TYK2) and their signal transduction mediated by IL-2.


Imeglimin

Imeglimin is a first-in-class oral agent which improves pancreatic beta-cell function and enhances insulin action in key tissues, including liver and skeletal muscle.


adult T-cell leukemia/lymphoma

A peripheral (mature) T-cell neoplasm linked to the human T-cell leukemia virus type 1 (HTLV-1), adult T-cell leukemia/lymphoma is endemic in several regions of the world, in particular Japan, the Caribbean, and parts of Central Africa.


Microsatellite instability-high colorectal cancer


classic Hodgkin lymphoma

Classical Hodgkin lymphoma (CHL) is a B-cell lymphoma characterized histologically by the presence of large mononuclear Hodgkin cells and multinucleated Reed-Sternberg (HRS) cells.


Klebsiella infectious disease

Infections with bacteria of the genus KLEBSIELLA.


Acinetobacter infectious disease

Infections with bacteria of the genus acinetobacter.


Exposure to radiation

Exposure to radioactive materials or ionizing radiation, whether by external irradiation, contact or contamination with radioactive material, or incorporation of radioactive materials, as in the case of certain diagnostic procedures.


Lower limb spasticity

Spasticity (velocity-dependent increase in tonic stretch reflexes with increased muscle tone and hyperexcitable tendon reflexes) in the muscles of the lower limbs, hips, and pelvis


AL amyloidosis

AL Amyloidosis is a plasma cell disorder characterized by the aggregation and deposition of insoluble amyloid fibrils derived from misfolding of monoclonal immunoglobulin light chains usually produced by a plasma cell tumor. It usually presents as primary systemic amyloidosis (PSA) with multiple organ involvement and less frequently as primary localized amyloidosis (PLA) restricted to a single organ.


Acute exacerbation of chronic congestive heart failure


esophageal squamous cell carcinoma

Esophageal squamous cell carcinoma (ESCC) is a type of esophageal carcinoma (EC) that can affect any part of the esophagus, but is usually located in the upper or middle third.


Autologous Hematopoietic Stem Cell Transplantation


Potassium Sulfate

Potassium sulfate is used to cleanse the colon (bowel) before colonoscopy in combination with sodium sulfate and magnesium sulfate.


Molidustat

Molidustat (BAY 85–3934) is a novel, orally bioavailable HIF-PH inhibitor that mimics hypoxia by stabilizing HIF-a subunits. Molidustat inhibits HIF-PH, allowing the accumulation of HIF, which then translocates to the nucleus where it activates the transcription of erythropoietin (EPO) and other hypoxia-inducible genes, thereby increasing endogenous EPO levels and formation of the red blood cell.


Anamorelin

Anamorelin is a potent and selective novel ghrelin receptor agonist that mimics the N-terminal active core of ghrelin. ANAM stimulates neuroendocrine responses and can induce rapid positive effects on appetite and metabolism, which can lead to an increase in body weight and lean body mass.


PABINAFUSP ALFA

Pabinafusp alfa is a fusion protein consisting of a humanized anti-hTfR antibody and hIDS. The humanized anti-hTfR antibody is considered to bind to TfRs expressed in the luminal cell membrane of brain microvascular endothelial cells composing the blood-brain barrier, being transported together with the TfR, passing through brain microvascular endothelial cells via transcytosis (which involves the mechanisms of uptake into the cell via endocytosis and release outside the cell via exocytosis) and further penetrating the basal lamina into neuronal cells in the brain parenchyma. In addition, after binding of pabinafusp alfa to M6PR on the cell membrane and cellular uptake via endocytosis, newly formed endosomes are thought to fuse with lysosomes and move into them. In patients with MPS, the type of accumulating GAG differs depending on the MPS phenotype, and CNS symptoms develops in patients with MPS phenotypes that are associated with the accumulation of HS. HS concentrations in CSF are high in patients with MPS-II who have intellectual disability, compared with those who do not have intellectual disability. These findings and other factors have suggested that HS plays a vital role in the development of CNS symptoms. The hIDS of pabinafusp alfa is considered to promote the degradation of HS in the brain parenchyma, thereby suppressing the development or progression of CNS symptoms. It has been demonstrated that pabinafusp alfa has binding affinity for M6PR and hTfR, and is taken up by human fibroblasts via the receptors.


DICLOFENAC ETALHYALURONATE SODIUM

Diclofenac etalhyaluronate is a 130,000 kDa compound in which the diclofenac molecule is attached via a 2-aminoethanol linker to the glucuronic acid moiety of the hyaluronic acid. Diclofenac is released through hydrolytic cleavage of the ester linkage and it inhibits the synthesis of prostaglandin E2.


osteoarthritis susceptibility 5

Any osteoarthritis in which the cause of the disease is a mutation in the GDF5 gene.


Esaxerenone

Esaxerenone is an oral, selective  nonsteroidal  mineralocorticoid  receptor antagonist used for the treatment of hypertension and diabetic nephropathies.


Mirogabalin besylate

Mirogabalin, a novel ligand for the alpha2delta subunit of voltage-gated calcium channels, has been approved for the treatment of peripheral neuropathic pain including painful diabetic peripheral neuropathy (DPNP) and postherpetic neuralgia (PHN) in Japan. Mirogabalin showed potent and selective binding affinities for the alpha2delta subunits, and slower dissociation rates for the alpha2delta-1 subunit than for the alpha2delta-2 subunit. It also showed potent and long-lasting analgesic effects in rat models of neuropathic pain, and wider safety margins for the central nervous system side effects. A pharmacological study using mutant mice demonstrated that the analgesic effects of mirogabalin were mediated by binding of the drug to the alpha2delta-1 subunit, not the alpha2delta-2 subunit.


POEMS syndrome

POEMS syndrome is a paraneoplastic syndrome characterized by polyradiculoneuropathy (P), organomegaly (O), endocrinopathy (E), clonal plasma cell disorder (M), and skin changes (S). Other features include papilledema, extravascular volume overload, sclerotic bone lesions, thrombocytosis/erythrocytosis, and elevated VEGF levels.


secondary progressive multiple sclerosis

A multiple sclerosis with a clinical course characterized by a progressive accumulation of neurological disability, independent of relapses, following an initial relapsing-remitting (RR) phase.


Anaplastic lymphoma kinase fusion oncogene positive non-small cell lung cancer


hordeolum

An acute, localized swelling of the eyelid that may be external or internal and usually is a pyogenic (typically staphylococcal) infection or abscess.


blepharitis

Inflammation of the eyelids near the eyelashes.


MELAS syndrome

MELAS (Mitochondrial myopathy, encephalopathy, lactic acidosis, and stroke) syndrome is a rare progressive multisystemic disorder characterized by encephalomyopathy, lactic acidosis, and stroke-like episodes. Other features include endocrinopathy, heart disease, diabetes, hearing loss, and neurological and psychiatric manifestations.


Sodium Selenite

The disodium salt of selenious acid. It is used therapeutically to supply the trace element selenium and is prepared by the reaction of SELENIUM DIOXIDE with SODIUM HYDROXIDE.

An inorganic sodium salt composed of sodium and selenite ions in a 2:1 ratio.


non-small cell squamous lung carcinoma

A squamous cell carcinoma that arises from the lung. It is characterized by the presence of large malignant cells. It includes the clear cell and papillary variants of squamous cell carcinoma.


familial amyloid neuropathy

Familial amyloid polyneuropathy (FAP) or transthyretin (TTR) amyloid polyneuropathy is a progressive sensorimotor and autonomic neuropathy of adulthood onset. Weight loss and cardiac involvement are frequent; ocular or renal complications may also occur.


atypical hemolytic-uremic syndrome

A rare, genetic thrombotic microangiopathy due to dysregulation of the alternative complement pathway and characterized by the triad of hemolytic anemia, thrombocytopenia, and acute renal dysfunction.


acute laryngopharyngitis

An upper respiratory tract disease which involves inflammation of both larynx and pharynx.


microscopic polyangiitis

Microscopic polyangiitis (MPA) is an inflammatory, necrotizing, systemic vasculitis that affects predominantly small vessels (i.e. small arteries, arterioles, capillaries, venules) in multiple organs.


Still's disease with juvenile onset and/or adult onset


cytokine release syndrome

A syndrome that occurs after therapeutic infusion of antibodies into the blood and is characterized by nausea, headache, tachycardia, hypotension, rash, and shortness of breath. It is caused by the release of cytokines from the cells that are targeted by the antibodies. Most patients experience a mild to moderate reaction; however, the reaction may be severe and life-threatening.


Pollakisuria

Increased frequency of urination.


Acute pyelonephritis caused by bacterium


Upacicalcet

Upacicalcet acts directly on parathyroid cell membrane calcium-sensing receptors and it suppresses excessive parathyroid hormone secretion.


Spiramycin A

A macrolide antibiotic produced by Streptomyces ambofaciens. The drug is effective against gram-positive aerobic pathogens, N. gonorrhoeae, and staphylococci. It is used to treat infections caused by bacteria and Toxoplasma gondii.

A macrolide antibiotic produced by various Streptomyces species that is used to treat toxoplasmosis and various other infections of soft tissues.


eosinophilic granulomatosis with polyangiitis

Eosinophilic granulomatosis with polyangiitis (EGPA), previously known as Churg-Strauss syndrome, is a systemic vasculitis of small-to medium vessels, characterized by asthma, transient pulmonary infiltrates, and hypereosinophilia.


pustulosis palmaris et plantaris

A chronic inflammatory disorder that generally occurs in adulthood and is characterized by sterile pustules intermingled with scaly erythemas, vesicles and hyperkeratosis occurring at distinct sites on the palms and soles. It is thought to be an autoimmune disorder precipitated by several factors, including local infection, and smoking.


Stress echocardiography using dobutamine


small intestine cancer

A primary or metastatic malignant neoplasm involving the small intestine.


Maralixibat

Maralixibat is a reversible inhibitor of the ileal bile acid transporter (IBAT). It decreases the reabsorption of bile acids (primarily the salt forms) from the terminal ileum. Pruritus is a common symptom in patients with ALGS and the pathophysiology of pruritus in patients with ALGS is not completely understood. Although the complete mechanism by which maralixibat improves pruritus in ALGS patients is unknown, it may involve inhibition of the IBAT, which results in decreased reuptake of bile salts, as observed by a decrease in serum bile acids.


Alagille syndrome

Alagille (AGS) syndrome is variably characterized by chronic cholestasis due to paucity of intrahepatic bile ducts, peripheral pulmonary artery stenosis, vertebrae segmentation anomalies, characteristic facies, posterior embryotoxon/anterior segment abnormalities, pigmentary retinopathy, and dysplastic kidneys.


acute respiratory failure

Life-threatening respiratory failure that develops rapidly. Causes include injury, sepsis, drug overdose, and pancreatitis. It manifests with dyspnea and cyanosis and may lead to cardiovascular shock.


Epiphysis closure

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a recombinant IL-2 product having a methionine added at the N-terminal.

An indolyl carbohydrate that is the beta-D-galactoside of 3-hydroxy-1H-indole in which the indole moiety is substituted at positions 4 and 5 by chlorine and bromine, respectively. It is used to test for the presence of an enzyme, beta-galactosidase, which cleaved the glycosidic bond to give 5-bromo-4-chloro-3-hydroxy-1H-indole, which immediately dimerises to give an intensely blue product.


IDURSULFASE BETA

Enzyme replacement therapy product for the treatment of Hunter syndrome


rectal cancer

A primary or metastatic malignant neoplasm that affects the rectum. Representative examples include carcinoma, lymphoma, and sarcoma.


indocyanine green acid form

A tricarbocyanine dye that is used diagnostically in liver function tests and to determine blood volume and cardiac output.


Recurrent Chronic Lymphoid Leukemia

The reemergence of chronic lymphocytic leukemia after a period of remission.


Nemorexant

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


Avacopan

Avacopan is a complement 5a receptor (C5aR) antagonist that inhibits the interaction between C5aR and the anaphylatoxin C5a. Avacopan blocks C5a-mediated neutrophil activation and migration. The precise mechanism by which avacopan exerts a therapeutic effect in patients with ANCA-associated vasculitis has not been definitively established.


Asciminib

Asciminib is an ABL/BCR-ABL1 tyrosine kinase inhibitor. Asciminib inhibits the ABL1 kinase activity of the BCRABL1 fusion protein, by binding to the ABL myristoyl pocket. In studies conducted in vitro or in animal models of CML, asciminib showed activity against wild-type BCR-ABL1 and several mutant forms of the kinase, including the T315I mutation.


Maribavir

Livtencity is an antiviral drug against human cytomegalovirus. The antiviral activity of maribavir is mediated by competitive inhibition of the protein kinase activity of human CMV enzyme pUL97, which results in inhibition of the phosphorylation of proteins. Maribavir inhibited wildtype pUL97 protein kinase in a biochemical assay with an IC50 value of 0.003 microM.


Pafolacianine

Cytalux is a fluorescent drug that targets folate receptor (FR) which may be overexpressed in ovarian cancer. Pafolacianine binds to FR-expressing cancer cells with affinity of approximately 1 nM, internalizes via receptor mediated endocytosis, and concentrates in FR-positive cancer tissues.


Inclisiran

Inclisiran is a double-stranded small interfering ribonucleic acid (siRNA), conjugated on the sense strand with triantennary N-Acetylgalactosamine (GalNAc) to facilitate uptake by hepatocytes. In hepatocytes, inclisiran utilizes the RNA interference mechanism and directs catalytic breakdown of mRNA for PCSK9. This increases LDL-C receptor recycling and expression on the hepatocyte cell surface, which increases LDL-C uptake and lowers LDL-C levels in the circulation.

Inclisiran is a long-acting, synthetic small interfering RNA (siRNA) directed against PCSK9. It is indicated for the treatment of primary hypercholesterolemia (familial and non-familial) or mixed dyslipidemia in adults.


EFGARTIGIMOD ALFA

Efgartigimod alfa-fcab is a human IgG1 antibody fragment that binds to the neonatal Fc receptor (FcRn), resulting in the reduction of circulating IgG.


TEZEPELUMAB

Tezepelumab-ekko is a thymic stromal lymphopoietin (TSLP) blocker, human monoclonal antibody IgG2lambda that binds to human TSLP with a dissociation constant of 15.8 pM and blocks its interaction with the heterodimeric TSLP receptor. TSLP is a cytokine mainly derived from epithelial cells and occupies an upstream position in the asthma inflammatory cascade. Airway inflammation is an important component in the pathogenesis of asthma. Multiple cell types (e.g., mast cells, eosinophils, neutrophils, macrophages, lymphocytes, ILC2 cells) and mediators (e.g., histamine, eicosanoids, leukotrienes, cytokines) are involved in airway inflammation. Blocking TSLP with tezepelumab-ekko reduces biomarkers and cytokines associated with inflammation including blood eosinophils, airway submucosal eosinophils, IgE, FeNO, IL-5, and IL-13; however, the mechanism of tezepelumab-ekko action in asthma has not been definitively established.


Immunosuppressive therapy for transplant


Monomethyl fumarate

The mechanism by which diroximel fumarate exerts therapeutic effects in MS is not fully understood. Diroximel fumarate acts via the major active metabolite, monomethyl fumarate. Preclinical studies indicate that the pharmacodynamic responses of monomethyl fumarate appears to be mediated, at least in part, through activation of the Nuclear factor (erythroid-derived 2)-like 2 (Nrf2) transcriptional pathway. Dimethyl fumarate has been shown to up regulate Nrf2-dependent antioxidant genes in patients.

A dicarboxylic acid monoester resulting from the formal condensation of one of the carboxy groups of fumaric acid with methanol. Is is a metabolite of dimethyl fumarate and used for the the treatment of patients with relapsing multiple sclerosis (MS). It also induces the NFE2L2 (Nrf2) transcription factor by  binding to KEAP1.


Difamilast

Difamilast is a novel atopic dermatitis drug that has phosphodiesterase 4 inhibitory activity. PDE4 inhibitors are believed to improve the symptoms of atopic dermatitis through suppression of the production of chemical mediators such as pro-inflammatory cytokines and through other anti-inflammatory effects.


lymphangioma

A benign lesion composed of dilated lymphatic channels. Painless swelling is the usual clinical manifestation.


Lymphangiomatosis

A lymphangioma affecting several anatomic sites.


Gorham-Stout disease

Gorham-Stout disease (GSD) is a rare disease of massive osteolysis associated with proliferation and dilation of lymphatic vessels. GSD may affect any bone in the body and can be monostotic or polyostotic. Symptoms at presentation are dependent upon the location(s) of the disease; the most common symptom is localized pain. The disease may be discovered after a pathological fracture.


lymphangiectasis

Dilatation of the lymphatic vessels.


ABROCITINIB

Abrocitinib is a Janus kinase (JAK)1 inhibitor. JAKs phosphorylate and activate Signal Transducers and Activators of Transcription (STATs) which modulate intracellular activity including gene expression. Inhibition of JAK1 modulates the signalling pathways by preventing the phosphorylation and activation of STATs. It is used for  treating adults with moderate to severe atopic dermatitis (also known as eczema, when the skin is itchy, red and dry).


systemic sclerosis

A chronic disorder, possibly autoimmune, marked by excessive production of collagen which results in hardening and thickening of body tissues. The two types of systemic scleroderma, limited cutaneous and diffuse cutaneous are classified with focus on the extent of affected skin. A relationship exists between the extent of skin area affected and degree of internal organ/system involvement. Systemic scleroderma can manifest itself in pulmonary fibrosis, Raynaud's syndrome, digestive system telangiectasias, renal hypertension and/or pulmonary hypertension.


Iobenguane (131I)

AZEDRA is an I 131 labeled iobenguane. Iobenguane is similar in structure to the neurotransmitter norepinephrine (NE) and is subject to the same uptake and accumulation pathways as NE. Iobenguane is taken up by the NE transporter in adrenergic nerve terminals and accumulates in adrenergically innervated tissues, such as the heart, lungs, adrenal medulla, salivary glands, liver, and spleen as well as tumors of neural crest origin. Pheochromocytoma and paraganglioma (PPGL) are tumors of neural crest origin that express high levels of the NE transporter on their cell surfaces. Following intravenous administration, AZEDRA is taken up and accumulates within pheochromocytoma and paraganglioma cells, and radiation resulting from radioactive decay of I 131 causes cell death and tumor necrosis.


paraganglioma

A benign or malignant neoplasm arising from paraganglia located along the sympathetic or parasympathetic nerves. Infrequently, it may arise outside the usual distribution of the sympathetic and parasympathetic paraganglia. Tumors arising from the adrenal gland medulla are called pheochromocytomas. Morphologically, paragangliomas usually display a nesting (Zellballen) growth pattern. There are no reliable morphologic criteria to distinguish between benign and malignant paragangliomas. The only definitive indicator of malignancy is the presence of regional or distant metastases.


SOTROVIMAB

Sotrovimab is a human IgG1 mAb that binds to a highly conserved epitope on the spike protein receptor binding domain of SARS-CoV-2.


REGDANVIMAB

Regdanvimab is a recombinant human IgG1 monoclonal antibody that binds to the receptor binding domain (RBD) of the spike(s) protein of SARS-CoV-2 consequently blocking cellular entry and SARS-CoV-2 infection.


Oteseconazole

Oteseconazole is an azole metalloenzyme inhibitor targeting the fungal sterol, 14alpha demethylase(CYP51), an enzyme that catalyzes an early step in the biosynthetic pathway of ergosterol, a sterol required for fungal cell membrane formation and integrity. Inhibition of CYP51 results in the accumulation of 14-methylated sterols, some of which are toxic to fungi. Through the inclusion of a tetrazole metal-binding group, oteseconazole has a lower affinity for human CYP enzymes.


SUTIMLIMAB

Sutimlimab-jome is an immunoglobulin G (IgG), subclass 4 (IgG4) monoclonal antibody (mAb) that inhibits the classical complement pathway (CP) and specifically binds to complement protein component 1, s subcomponent (C1s), a serine protease which cleaves C4. Sutimlimab-jome does not inhibit the lectin and alternative pathways. Inhibition of the classical complement pathway at the level of C1s prevents deposition of complement opsonins on the surface of RBCs, resulting in inhibition of hemolysis in patients with CAD.


TEBENTAFUSP

Tebentafusp-tebn is a bispecific gp100 peptide-HLA-A02:01 directed T cell receptor CD3 T cell engager. The TCR arm binds to a gp100 peptide presented by human leukocyte antigen-A*02:01 (HLA¬ A02:01) on the cell surface of uveal melanoma tumor cells. In vitro, tebentafusp-tebn bound to HLA-A02:01-positive uveal melanoma cells and activated polyclonal T cells to release inflammatory cytokines and cytolytic proteins, which results in direct lysis of uveal melanoma tumor cells.


Unresectable Uveal Melanoma

Uveal melanoma that is not amenable to surgical resection.


Metastatic Uveal Melanoma

Uveal melanoma that has spread from its primary site to another anatomic site.


RELATLIMAB

Relatlimab is a human IgG4 monoclonal antibody that binds to the LAG-3 receptor, blocks interaction with its ligands, including MHC II, and reduces LAG-3 pathway-mediated inhibition of the immune response. Antagonism of this pathway promotes T cell proliferation and cytokine secretion. Binding of the PD-1 ligands, PD-L1 and PD-L2, to the PD-1 receptor found on T cells, inhibits T-cell proliferation and cytokine production. Upregulation of PD-1 ligands occurs in some tumors, and signaling through this pathway can contribute to inhibition of active T-cell immune surveillance of tumors. Nivolumab is a human IgG4 monoclonal antibody that binds to the PD-1 receptor, blocks interaction with its ligands PD-L1 and PD-L2, and reduces PD-1 pathwaymediated inhibition of the immune response, including the anti-tumor immune response. In syngeneic mouse tumor models, blocking PD-1 activity resulted in decreased tumor growth. The combination of nivolumab (anti-PD-1) and relatlimab (anti-LAG-3) results in increased T-cell activation compared to the activity of either antibody alone. In murine syngeneic tumor models, LAG-3 blockade potentiates the anti-tumor activity of PD-1 blockage, inhibiting tumor growth and promoting tumor regression.


FARICIMAB

Faricimab is a humanized bispecific antibody that acts through inhibition of two pathways by binding to VEGF-A and Ang-2. By inhibiting VEGF-A, faricimab suppresses endothelial cell proliferation, neovascularization and vascular permeability. By inhibiting Ang-2, faricimab is thought to promote vascular stability and desensitize blood vessels to the effects of VEGF-A. Ang-2 levels are increased in some patients with nAMD and DME. The contribution of Ang-2 inhibition to the treatment effect and clinical response for nAMD and DME has yet to be established.


Infection of skin of eyelid and periocular region


TIRZEPATIDE

Tirzepatide is a GIP receptor and GLP-1 receptor agonist. It is a 39-amino-acid modified peptide with a C20 fatty diacid moiety that enables albumin binding and prolongs the half-life. Tirzepatide selectively binds to and activates both the GIP and GLP-1 receptors, the targets for native GIP and GLP-1. Tirzepatide enhances first- and second-phase insulin secretion, and reduces glucagon levels, both in a glucosedependent manner.


SOMATROGON

Somatrogon is a glycoprotein comprised of the amino acid sequence of hGH with one copy of the of C-terminal peptide (CTP) from the beta chain of human chorionic gonadotropin (hCG) at the N-terminus and two copies of CTP (in tandem) at the C-terminus. The glycosylation and CTP domains account for the half-life of somatrogon, which allows for weekly dosing. Somatrogon binds to the GH receptor and initiates a signal transduction cascade culminating in changes in growth and metabolism. Consistent with GH signalling, somatrogon binding leads to activation of the STAT5b signalling pathway and increases the serum concentration of IGF-1. IGF-1 was found to increase in a dose-dependent manner during treatment with somatrogon partially mediating the clinical effect. As a result, GH and IGF-1 stimulate metabolic changes, linear growth and enhance growth velocity in paediatric patients with GHD.


Clazosentan

Clazosentan is an endothelin receptor antagonist indicated for the prevention of cerebrovascular spasm after surgery for subarachnoid hemorrhage caused by cerebral aneurysm, and cerebral infarctions and cerebral ischemic symptoms accompanying the cerebrovascular spasm.


Gefapixant

Gefapixant is a selective, non-narcotic P2X3 receptor antagonist being approved for the treatment of refractory chronic cough or unexplained chronic cough.


Nirmatrelvir

Nirmatrelvir plus ritonavir (Paxlovid) is an antiviral therapeutic developed by Pfizer for the treatment and post-exposure prophylaxis of coronavirus disease 2019 (COVID-19). It received conditional approval  by EMA, FDA and PMDA. Nirmatrelvir (PF-07321332) is a peptidomimetic inhibitor of the SARS-CoV-2 main protease (Mpro), also referred to as 3C-like protease (3CLpro) or nsp5 protease. Inhibition of the SARS-CoV-2 Mpro renders the protein incapable of processing polyprotein precursors which leads to the prevention of viral replication. Ritonavir inhibits the CYP3A-mediated metabolism of PF-07321332, thereby providing increased plasma concentrations of PF-07321332.

An azabicyclohexane that is (1R,5S)-3-azabicyclo[3.1.0]hexane substituted by {(1S)-1-cyano-2-[(3S)-2-oxopyrrolidin-3-yl]ethyl}aminoacyl, 3-methyl-N-(trifluoroacetyl)-L-valinamide, methyl and methyl groups at positions 2S, 3, 6 and 6, respectively. It is the first orally administered inhibitor of SARS-CoV-2 main protease developed by Pfizer and used in combination with ritonavir for the treatment of COVID-19.


Tapinarof is an aryl hydrocarbon receptor (AhR) agonist. The specific mechanisms by which VTAMA cream exerts its therapeutic action in psoriasis patients are unknown.


monkeypox

An infection that is caused by an Orthopoxvirus, which is transmitted by primates or rodents, and which is characterized by a prodromal syndrome of fever, chills, headache, myalgia, and lymphedema; initial symptoms are followed by a generalized papular rash that typically progresses from vesiculation through crusting over the course of two weeks.


cowpox

A mild, eruptive skin disease of milk cows caused by cowpox virus, with lesions occurring principally on the udder and teats. Human infection may occur while milking an infected animal.


Mitapivat is a pyruvate kinase activator that acts by allosterically binding to the pyruvate kinase tetramer and increasing pyruvate kinase (PK) activity. The red blood cell (RBC) form of pyruvate kinase (PK-R) is mutated in PK deficiency, which leads to reduced adenosine triphosphate(ATP), shortened RBC lifespan, and chronic hemolysis.


pyruvate kinase deficiency of red cells

Hemolytic anemia due to red cell pyruvate kinase (PK) deficiency is a metabolic disorder characterized by a variable degree of chronic nonspherocytic hemolytic anemia.


Pacritinib

Pacritinib is a kinase inhibitor with activity against Janus-associated kinase 2 (JAK2) and Fms-like receptor tyrosine kinase 3 (FLT3). Pacritinib inhibits both wild-type JAK2 and the JAK2V617F mutant form that is common in patients with myeloproliferative neoplasms. Pacritinib also exhibits inhibitory activity against additional cellular kinases (such as colony-stimulating factor 1 and interleukin 1 receptor-associated kinase 1), the clinical relevance of which is unknown.


Ganaxolone

The precise mechanism by which ganaxolone exerts its therapeutic effects in the treatment of seizures associated with CDD is unknown, but its anticonvulsant effects are thought to result from positive allosteric modulation of the gamma-aminobutyric acid type A (GABAA) receptor in the CNS.


developmental and epileptic encephalopathy, 2

Any early infantile epileptic encephalopathy in which the cause of the disease is a mutation in the CDKL5 gene.


PSMA-617 Lu-177

Lutetium Lu 177 vipivotide tetraxetan is a radioligand therapeutic agent. The active moiety of lutetium Lu 177 vipivotide tetraxetan is the radionuclide lutetium-177 which is linked to a moiety that binds to PSMA, a transmembrane protein that is expressed in prostate cancer, including mCRPC. Upon binding of lutetium Lu 177 vipivotide tetraxetan to PSMA- expressing cells, the beta-minus emission from lutetium-177 delivers radiation to PSMA-expressing cells, as well as to surrounding cells, and induces DNA damage which can lead to cell death.


Mavacamten

Mavacamten is an allosteric and reversible inhibitor selective for cardiac myosin. Mavacamten modulates the number of myosin heads that can enter “on actin” (power-generating) states, thus reducing the probability of force-producing (systolic) and residual (diastolic) cross-bridge formation. Excess myosin actin cross-bridge formation and dysregulation of the super-relaxed state are mechanistic hallmarks of HCM. Mavacamten shifts the overall myosin population towards an energy-sparing, recruitable, super-relaxed state. In HCM patients, myosin inhibition with mavacamten reduces dynamic LVOT obstruction and improves cardiac filling pressures.


eosinophilic esophagitis

Eosinophilic esophagitis (EoE) is a chronic, allergic disease of the esophagus characterized clinically by symptoms of esophageal dysfunction (including vomiting, dysphagia, feeding disorders, food impaction and abdominal pain) which persist after treatment with proton pump inhibitors (PPIs).


salivary gland cancer

A primary or metastatic malignant neoplasm that affects the major or minor salivary glands. Representative examples include carcinoma, lymphoma, and sarcoma.


pemphigus foliaceus

Pemphigus foliaceous is a rare superficial pemphigus disease characterized by multiple, pruritic, scaly, crusted cutaneous erosions, with flaky circumscribed patches, localized mostly on the face, scalp, trunk and extremities, often presenting an erythematous base. Mucosal involvement is rarely observed.


Molnupiravir

EIDD-1931 has broad spectrum antiviral activity against SARS-CoV-2, MERS-CoV, SARS-CoV, and related zoonotic group 2b or 2c Bat-CoVs, as well as increased potency against a coronavirus bearing resistance mutations to the nucleoside analog inhibitor remdesivir

A nucleoside analogue that is N(4)-hydroxycytidine in which the 5'-hydroxy group is replaced by a (2-methylpropanoyl)oxy group. It is the prodrug of the active antiviral ribonucleoside analog N(4)-hydroxycytidine (EIDD-1931), has activity against a number of RNA viruses including SARS-CoV-2, MERS-CoV, and seasonal and pandemic influenza viruses. It is currently in phase III trials for the treatment of patients with COVID-19.


SPESOLIMAB

Spesolimab-sbzo is a humanized monoclonal immunoglobulin G1 antibody that inhibits interleukin-36 (IL-36) signaling by specifically binding to the IL36R. Binding of spesolimab-sbzo to IL36R prevents the subsequent activation of IL36R by cognate ligands (IL-36 alpha, beta and gamma) and downstream activation of pro inflammatory and pro-fibrotic pathways. The precise mechanism linking reduced IL36R activity and the treatment of flares of GPP is unclear.


generalized pustular psoriasis

A rare and extreme form of psoriasis characterized by the appearance of sterile pustules which can take many patterns. All the main pathological features of the disease are accentuated. Generalized pustular psoriasis is clinically heterogeneous in its age at onset, precipitants, severity, and natural history. Many overlapping clinical entities are recognized. There is a relationship between these entities and plaque psoriasis, as some individuals may have episodes of plaque psoriasis preceding or following the generalized pustular psoriasis, but in others generalized pustular psoriasis occurs as the sole phenotype without plaque psoriasis at any time.


Deucravacitinib

Deucravacitinib is an inhibitor of tyrosine kinase 2 (TYK2). TYK2 is a member of the Janus kinase (JAK) family. Deucravacitinib binds to the regulatory domain of TYK2, stabilizing an inhibitory interaction between the regulatory and the catalytic domains of the enzyme. This results in allosteric inhibition of receptor-mediated activation of TYK2 and its downstream activation of Signal Transducers and Activators of Transcription (STATs) as shown in cell-based assays. JAK kinases, including TYK2, function as pairs of homo- or heterodimers in the JAK-STAT pathways. TYK2 pairs with JAK1 to mediate multiple cytokine pathways and also pairs with JAK2 to transmit signals as shown in cell-based assays. The precise mechanism linking inhibition of TYK2 enzyme to therapeutic effectiveness in the treatment of adults with moderate-to-severe plaque psoriasis is not currently known.


Terlipressin

Terlipressin is a synthetic vasopressin analogue with twice the selectivity for vasopressin V1 receptors versus V2 receptors. Terlipressin acts as both a prodrug for lysine-vasopressin, as well as having pharmacologic activity on its own. Terlipressin is thought to increase renal blood flow in patients with hepatorenal syndrome by reducing portal hypertension and blood circulation in portal vessels and increasing effective arterial volume and mean arterial pressure (MAP). It is used to control bleeding of ESOPHAGEAL VARICES and for the treatment of HEPATORENAL SYNDROME.


ischemic disease

Lack of blood supply to an area of the body, resulting in impairment of tissue oxygenation.


EFLAPEGRASTIM

Eflapegrastim-xnst is a recombinant human granulocyte growth factor that binds to G-CSF receptors on myeloid progenitor cells and neutrophils, triggering signaling pathways that control cell differentiation, proliferation, migration and survival.


Febrile Neutropenia

Neutropenia associated with fever, the latter indicating the presence of an infection.


Tauroursodeoxycholic acid

Relyvrio contains two active ingredients: sodium phenylbutyrate and taurursodiol, being indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults.The mechanism by which relyvrio exerts its therapeutic effects in patients with ALS is unknown.

A bile acid taurine conjugate derived from ursoodeoxycholic acid.


Futibatinib

Futibatinib is a small molecule kinase inhibitor of FGFR 1, 2, 3, and 4 with IC50 values of less than 4 nM. Futibatinib covalently binds FGFR. Constitutive FGFR signaling can support the proliferation and survival of malignant cells. Futibatinib inhibited FGFR phosphorylation and downstream signaling and decreased cell viability in cancer cell lines with FGFR alterations including FGFR fusions/rearrangements, amplifications, and mutations. Futibatinib demonstrated anti-tumor activity in mouse and rat xenograft models of human tumors with activating FGFR genetic alterations.


intrahepatic cholangiocarcinoma

A carcinoma that arises from the intrahepatic bile duct epithelium in any site of the intrahepatic biliary tree. Grossly, the malignant lesions are solid, nodular, and grayish. Morphologically, the vast majority of cases are adenocarcinomas. Signs and symptoms include malaise, weight loss, right upper quadrant abdominal pain, and night sweats. Early detection is difficult and the prognosis is generally poor.


ELADOCAGENE EXUPARVOVEC

Eladocagene exuparvovec is a gene therapy medicinal product that expresses the human aromatic L-amino acid decarboxylase enzyme (hAADC). It is a non-replicating recombinant adeno-associated virus serotype 2 (AAV2) based vector containing the cDNA of the human dopa decarboxylase (DDC) gene under the control of the cytomegalovirus immediate-early promoter.


aromatic L-amino acid decarboxylase deficiency

Aromatic L-amino acid decarboxylase deficiency is a very rare, severe, genetic neurometabolic disorder associated with clinical manifestations related to underproduction of serotonin and dopamine, mainly hypotonia, hypokinesia, ptosis oculogyric crises, and signs of autonomic dysfunction.


Gadopiclenol

Gadopiclenol is a paramagnetic molecule (macrocyclic non-ionic complex of gadolinium) that develops a magnetic moment when placed in a magnetic field. The magnetic moment alters the relaxation rates of water protons in its vicinity in the body, leading to an increase in signal intensity (brightness) of tissues.


Medicinal product acting as imaging agent (product)


Sodium Bicarbonate

A white, crystalline powder that is commonly used as a pH buffering agent, an electrolyte replenisher, systemic alkalizer and in topical cleansing solutions.


Thallium chloride

Lutetium (177 Lu) chloride is a radiopharmaceutical precursor, and it is not intended for direct use in patients. It is to be used only for the radiolabelling of carrier molecules that have been specifically developed and authorised for radiolabelling with lutetium (177 Lu) chloride.


TIXAGEVIMAB

Tixagevimab is a recombinant human IgG1 monoclonal antibody used in combination with cilgavimab for the treatment of COVID-19.


CILGAVIMAB

Cilgavimab is a recombinant human IgG1 monoclonal antibody used in combination with tixagevimab for the treatment of COVID-19.


Darinaparsin

Darinaparsin, an organoarsenic compound with anticancer activity, is a novel mitochondrial-targeted agent being developed for the treatment of various hematologic and solid tumors. The proposed mechanism of action of the drug involves the disruption of mitochondrial function, increased production of reactive oxygen species, and modulation of intracellular signal transduction pathways.


Pimitespib is an oral small molecule inhibitor of the alpha and beta isoforms of heat shock protein 90 (HSP90).


Ferric derisomaltose is a complex of iron (III) hydroxide and derisomaltose, an iron carbohydrate oligosaccharide that releases iron. Iron binds to transferrin for transport to erythroid precursor cells to be incorporated into hemoglobin.


Carotegrast methyl

Carotegrast methyl is a small-molecule alpha4 integrin antagonist which received its first approval in Japan for the treatment of moderate ulcerative colitis in patients who had inadequate response to 5-aminosalicylic acid. The active metabolite of carotegrast methyl exerts an anti-inflammatory effect by blocking the interaction of alpha4beta1 or alpha4beta7 integrins and their ligands, VCAM-1 and MAd-CAM-1, thereby inhibiting the adhesion of inflammatory cells, including T cells, to vascular endothelial cells and extravasation into inflammatory sites.


NEMOLIZUMAB

Nemolizumab is a subcutaneously administered humanized anti-interleukin-31 (IL-31) receptor A (IL-31RA) monoclonal antibody to treat skin diseases, including atopic dermatitis, AD associated pruritus, prurigo nodularis, chronic kidney disease associated pruritus and systemic sclerosis.


TREMELIMUMAB

CTLA-4 is a negative regulator of T-cell activity. Tremelimumab-actl is a monoclonal antibody that binds to CTLA-4 and blocks the interaction with its ligands CD80 and CD86, releasing CTLA-4-mediated inhibition of T-cell activation. In synergistic mouse tumor models, blocking CTLA-4 activity resulted in decreased tumor growth and increased proliferation of T cells in tumors.


Anaplastic lymphoma kinase fusion oncogene negative non-small cell lung cancer


Mirvetuximab soravtansine

Mirvetuximab soravtansine-gynx is an antibody-drug conjugate (ADC). The antibody is a chimeric IgG1 directed against folate receptor alpha (FRalpha). The small molecule, DM4, is a microtubule inhibitor attached to the antibody via a cleavable linker. Upon binding to FRalpha, mirvetuximab soravtansine-gynx is internalized followed by intracellular release of DM4 via proteolytic cleavage. DM4 disrupts the microtubule network within the cell, resulting in cell cycle arrest and apoptotic cell death.


TEPLIZUMAB

Teplizumab-mzwv binds to CD3 (a cell surface antigen present on T lymphocytes) and delays the onset of Stage 3 type 1 diabetes in adults and pediatric patients aged 8 years and older with Stage 2 type 1 diabetes. The mechanism may involve partial agonistic signaling and deactivation of pancreatic beta cell autoreactive T lymphocytes. Teplizumab-mzwv leads to an increase in the proportion of regulatory T cells and of exhausted CD8+ T cells in peripheral blood.


Olutasidenib

Olutasidenib is a small-molecule inhibitor of mutated isocitrate dehydrogenase-1 (IDH1). In patients with AML, susceptible IDH1 mutations are defined as those leading to increased levels of 2- hydroxyglutarate (2-HG) in the leukemia cells and where efficacy is predicted by 1) clinically meaningful remissions with the recommended dose of olutasidenib and/or 2) inhibition of mutant IDH1 enzymatic activity at concentrations of olutasidenib sustainable at the recommended dosage according to validated methods. The most common of such mutations in patients with AML are R132H and R132C substitutions. In vitro, olutasidenib inhibited mutated IDH1 R132H, R132L, R132S, R132G, and R132C proteins; wild-type IDH1 or mutated IDH2 proteins were not inhibited. Olutasidenib inhibition of mutant IDH1 led to decreased 2-HG levels in vitro and in in vivo xenograft models.


Refractory acute myeloid leukemia

Acute myeloid leukemia that does not respond to treatment.


Adagrasib

Adagrasib is an irreversible inhibitor of KRAS G12C that covalently binds to the mutant cysteine in KRAS G12C and locks the mutant KRAS protein in its inactive state that prevents downstream signaling without affecting wild-type KRAS protein. Adagrasib inhibits tumor cell growth and viability in cells harboring KRAS G12C mutations and results in tumor regression in KRAS G12C-mutated tumor xenograft models with minimal off-target activity.


xenon (129Xe) hyperpolarized

When hyperpolarized xenon Xe 129 gas is inhaled, a multi-nuclear capable MRI scanner can be used to image the hyperpolarized xenon Xe 129 distribution throughout the ventilated lung. Hyperpolarized xenon Xe 129 nuclei are directly detected by a Xe 129 MRI coil.


Debrase

NexoBrid (anacaulase-bcdb) dissolves burn wound eschar. The specific components responsible for this effect have not been identified.


Skin eschar

A crust that covers necrotic tissue in the skin that is caused by a burn or gangrene.


UBLITUXIMAB

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


VUTRISIRAN

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


MOSUNETUZUMAB

Mosunetuzumab is an anti-CD20/CD3 T-cell engaging bispecific antibody targeting CD20-expressing B-cells. It is a conditional agonist; targeted B-cell killing is observed only upon simultaneous binding to CD20 on B-cells and CD3 on T-cells. Engagement of both arms of mosunetuzumab results in the formation of an immunologic synapse between a target B cell and a cytotoxic T cell leading to T-cell activation. Subsequent directed release of perforin and granzymes from T-cell activation through the immunologic synapsis induce B-cell lysis leading to cell death.


Linzagolix

Linzagolix is a selective, non-peptide gonadotropin-releasing hormone (GnRH) receptor antagonist that inhibits endogenous GnRH signalling by binding competitively to GnRH receptors in the pituitary gland, thereby modulating the hypothalamic-pituitary-gonadal axis.


Betula pendula bark extract

A dry extract from two species of birch bark consisting of naturally occurring substances known as triterpenes, including betulin, betulinic acid, erythrodiol, lupeol and oleanolic acid. It is used in adults and children aged 6 months or older with epidermolysis bullosa but the precise mechanism of action in wound healing is not known.


OLIPUDASE ALFA

Olipudase alfa is a recombinant human acid sphingomyelinase that reduces sphingomyelin (SM) accumulation in organs of patients with Acid Sphingomyelinase Deficiency (ASMD).


Niemann-Pick disease type A

Niemann-Pick disease type A is a very severe subtype of Niemann-Pick disease, an autosomal recessive lysosomal disease, and is characterized clinically by onset in infancy or early childhood with failure to thrive, hepatosplenomegaly, and rapidly progressive neurodegenerative disorders.


Niemann-Pick disease type B

Niemann-Pick disease type B is a mild subtype of Niemann-Pick disease, an autosomal recessive lysosomal disease, and is characterized clinically by onset in childhood with hepatosplenomegaly, growth retardation, and lung disorders such as infections and dyspnea


Lenacapavir

Lenacapavir is a multistage, selective inhibitor of HIV-1 capsid function that directly binds to the interface between capsid protein (CA) subunits. Lenacapavir inhibits HIV-1 replication by interfering with multiple, essential steps of the viral lifecycle, including capsid-mediated nuclear uptake of HIV-1 proviral DNA (by blocking nuclear import proteins binding to capsid), virus assembly and release (by interfering with Gag/Gag-Pol functioning, reducing production of CA subunits), and capsid core formation (by disrupting the rate of capsid subunit association, leading to malformed capsids).


VALOCTOCOGENE ROXAPARVOVEC

Valoctocogene roxaparvovec is a gene therapy medicinal product that expresses the B-domain deleted SQ form of human coagulation factor VIII (hFVIII-SQ). It is a non-replicating recombinant adeno-associated virus serotype AAV5 based vector containing the cDNA of the B-domain deleted SQ form of human coagulation factor VIII gene under the control of a liver-specific promoter.


severe hemophilia A

Severe hemophilia A is a form of hemophilia A characterized by a large deficiency of factor VIII leading to frequent spontaneous hemorrhage and abnormal bleeding as a result of minor injuries, or following surgery or tooth extraction.


Hepatic fibrosis

The presence of excessive fibrous connective tissue in the liver. Fibrosis is a reparative or reactive process.


TECLISTAMAB

Teclistamab is a full-size, IgG4-PAA bispecific antibody that targets the CD3 receptor expressed on the surface of T cells and B cell maturation antigen (BCMA), which is expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B cells and plasma cells. With its dual binding sites, teclistamab is able to draw CD3+ T cells in close proximity to BCMA+ cells, resulting in T cell activation and subsequent lysis and death of BCMA+ cells, which is mediated by secreted perforin and various granzymes stored in the secretory vesicles of cytotoxic T cells. This effect occurs without regard to T cell receptor specificity or reliance on major histocompatibility complex (MHC) Class 1 molecules on the surface of antigen presenting cells.


Helicobacter pylori infection

A recurrent infection of the GI tract with helicobacter pylori, a gram-negative, microaerophilic bacterium usually found in the stomach.


IgA glomerulonephritis

Inflammation of a specific segment of glomeruli within the kidney.


amebic dysentery

Dysentery caused by intestinal amebic infection, chiefly with entamoeba histolytica. This condition may be associated with amebic infection of the liver and other distant sites.


Sepsis caused by methicillin resistant Staphylococcus aureus


Methicillin resistant Staphylococcus aureus skin infection


Methicillin-resistant Staphylococcus aureus infection

Infection with staphylococcus aureus resistant to the antibiotic methicillin (MRSA). MRSA can infect any individual but is more common among hospitalized patients, and can also occur as an opportunistic infection.


Low back pain

An unpleasant sensation characterized by physical discomfort (such as pricking, throbbing, or aching) localized to the lower back.


Menopausal syndrome

A grouping of variable physical, vasomotor and psychological symptoms in climacteric females. Physical symptoms include: cessation of menses, headaches, fatigue, weight gain and vaginal dryness. Vasomotor symptoms typically include: palpitations, hot flashes and night sweats. Psychological symptoms may include: decrease in libido, emotional lability, difficulty concentrating and insomnia.


ovarian dysfunction

The inability of the ovaries to function.


Dydrogesterone

A synthetic progestational hormone with no androgenic or estrogenic properties. Unlike many other progestational compounds, dydrogesterone produces no increase in temperature and does not inhibit OVULATION.


TALC

Finely powdered native hydrous magnesium silicate. It is used as a dusting powder, either alone or with starch or boric acid, for medicinal and toilet preparations. It is also an excipient and filler for pills, tablets, and for dusting tablet molds. (From Merck Index, 11th ed)


inborn error of immunity

A disorder in which the immune system is unable to mount an adequate immune response.


Hypoproteinemia

A decreased concentration of protein in the blood.


Metastatic malignant neoplasm to bone

The spread of a malignant neoplasm from a primary site to the skeletal system.  The majority of metastatic neoplasms to the bone are carcinomas.


INDIGOTINDISULFONATE

Indolesulfonic acid used as a dye in renal function testing for the detection of nitrates and chlorates, and in the testing of milk. It is a dye excreted by the kidney through tubular secretion and enhances visualization of the ureteral orifices by its deep blue color.

A member of the class of indolones obtained by formal 2,2'-oxidative coupling of two molecules of 3-oxo-2,3-dihydroindole-5-sulfonic acids.


diagnostic cystoscopy revealed bullous edema of both ureteral orifices


mantle cell lymphoma

Mantle cell lymphoma is a rare form of malignant non-Hodgkin lymphoma affecting B lymphocytes in the lymph nodes in a region called the ``mantle zone''.


migraine with aura

A migraine disorder characterized by episodes that are preceded by focal neurological symptoms.


Infection due to carbapenem resistant Acinetobacter


thyroid gland undifferentiated (anaplastic) carcinoma

A primary carcinoma of the thyroid gland composed of undifferentiated cells. The malignant cells demonstrate evidence of epithelial differentiation, either by immunohistochemistry or electron microscopic studies. Microscopically, in the majority of cases there is a mixture of spindle, epithelioid, and giant cells. The vast majority of the patients present with a rapidly enlarging neck mass. The clinical course is usually aggressive.


Nonexudative age-related macular degeneration


Solid carcinoma

A carcinoma morphologically characterized by the presence of solid sheets of malignant epithelial cells in tissues.


chronic liver failure

Liver failure that develops slowly and gradually for some time, possibly for years, often as the result of cirrhosis, or malnutrition.


thyroid gland oncocytic follicular carcinoma

A follicular carcinoma of the thyroid gland, characterized by the presence of large cells with eosinophilic granular cytoplasm and pleomorphic nuclei with prominent, eosinophilic nucleoli. The nuclear features that characterize the papillary carcinomas of the thyroid gland are absent.


acute graft versus host disease

A syndrome of immunologically mediated tissue damage that may occur following an allogeneic transplant, usually affecting the skin, liver, and GI tract. The onset is usually within one hundred days of transplantation or immunologic manipulation.


Splenomegaly

Abnormal increased size of the spleen.


Metastatic Colon Carcinoma

A carcinoma that arises from the colon and has metastasized to another anatomic site.


Metastatic basal cell carcinoma

A carcinoma that arises from the basal cells and has metastasized to another anatomic site.


Non-small cell lung cancer without mutation in epidermal growth factor receptor


molluscum contagiosum

A common, benign, usually self-limited viral infection of the skin and occasionally the conjunctivae by a poxvirus (molluscum contagiosum virus). (Dorland, 27th ed)


Lotilaner

Lotilaner is a gamma-aminobutyric acid (GABA)-gated chloride channel inhibitor selective for mites. Inhibition of these GABA chloride channels causes a paralytic action in the target organism leading to its death. Lotilaner is not an inhibitor of mammalian GABA mediated chloride channels when tested at up to 30 microM (18 microg/mL) in vitro (approximately 1100 times the RHOD).


NIRSEVIMAB

Nirsevimab is a recombinant neutralising human IgG1kappa long-acting monoclonal antibody to the prefusion conformation of the RSV F protein which has been modified with a triple amino acid substitution (YTE) in the Fc region to extend serum half-life. Nirsevimab binds to a highly conserved epitope in antigenic site OE on the prefusion protein with dissociation constants Kd = 0.12 nM and Kd = 1.22 nM for RSV subtype A and B strains, respectively. Nirsevimab inhibits the essential membrane fusion step in the viral entry process, neutralising the virus and blocking cell-to-cell fusion.


Metastatic malignant neoplasm to prostate

The spread of a malignant neoplasm to the prostate gland from an adjacent or distant anatomic site.


acute myeloblastic leukemia without maturation

An acute myeloid leukemia (AML) characterized by blasts without evidence of maturation to more mature neutrophils. (WHO, 2001)


CHORIONIC GONADOTROPIN

A gonadotropic glycoprotein hormone produced primarily by the PLACENTA. Similar to the pituitary LUTEINIZING HORMONE in structure and function, chorionic gonadotropin is involved in maintaining the CORPUS LUTEUM during pregnancy. CG consists of two noncovalently linked subunits, alpha and beta. Within a species, the alpha subunit is virtually identical to the alpha subunits of the three pituitary glycoprotein hormones (TSH, LH, and FSH), but the beta subunit is unique and confers its biological specificity (CHORIONIC GONADOTROPIN, BETA SUBUNIT, HUMAN).


CHORIOGONADOTROPIN ALFA

Choriogonadotropin alfa, is an analogue of Luteinizing Hormone (LH) and binds to the LH/hCG receptor of the granulosa and theca cells of the ovary to effect these changes in the absence of an endogenous LH surge. In pregnancy, hCG, secreted by the placenta, maintains the viability of the corpus luteum to provide the continued secretion of estrogen and progesterone necessary to support the first trimester of pregnancy.


acute pyelonephritis

Sudden onset pyelonephritis.


B-cell acute lymphoblastic leukemia

A neoplasm of lymphoblasts committed to the B-cell lineage, typically composed of small to medium-sized blast cells. When the neoplasm involves predominantly the bone marrow and the peripheral blood, it is called B acute lymphoblastic leukemia. When it involves nodal or extranodal sites, it is called B lymphoblastic lymphoma. (WHO, 2001)


prostate carcinoma

A carcinoma that arises from epithelial cells of the prostate gland.


Malignant neoplasm of cardioesophageal junction of stomach


adult-onset Still disease

A rare inflammatory multisystem disorder characterized clinically by fever of unknown origin, arthralgia or arthritis, hyperleucocytosis, and typical skin rash.


Familial Amyloid Neuropathy, Portuguese Type


Bardet-Biedl syndrome

A ciliopathy with multisystem involvement. It is invariantly characterized by rod-cone dystrophy, and at least three additional non-ocular features such as intellectual disability, obesity, polydactyly, hypogonadism, or renal anomalies as primary manifestations. In the absence of one of these four primary clinical features, the diagnosis of BBS is made when at least two secondary features are observed, including hepatic fibrosis, diabetes mellitus, reproductive and developmental abnormalities, growth retardation, speech delays, or cardiovascular problems


fetal cytomegalovirus syndrome

An infection with the Cytomegalovirus that is present from birth.


Disorder due to type 2 diabetes mellitus


hypercholesterolemia, familial, 1

An autosomal dominant inherited disorder characterized by very high levels of low-density lipoprotein cholesterol (LDL-C) and total cholesterol in the blood. It is usually caused by mutations in the LDLR gene which is located on the short arm of chromosome 19.


myeloid neoplasm associated with FGFR1 rearrangement

Hematologic neoplasms characterized by the rearrangement of the FGFR1 gene, resulting in translocations with an 8p11 breakpoint. Patients present with a myeloproliferative neoplasm, acute myeloid leukemia, lymphoblastic lymphoma/leukemia of T or B-cell lineage, or acute leukemia of mixed phenotype.


High grade squamous intraepithelial neoplasia of esophagus


histiocytic sarcoma

An aggressive malignant neoplasm with a poor response to therapy, usually presenting as stage III/IV disease. It is characterized by the presence of neoplastic cells with morphologic and immunophenotypic characteristics similar to those seen in mature histiocytes.


History of myocardial infarction due to atherothrombotic coronary artery disease


alkaptonuria

A metabolic disease characterized by the accumulation of homogentisic acid (HGA) and its oxidized product, benzoquinone acetic acid (BQA), in various tissues (e.g. cartilage, connective tissue) and body fluids (urine, sweat), causing urine to darken when exposed to air as well as grey-blue coloration of the sclera and ear helix (ochronosis), and a disabling joint disease involving both the axial and peripheral joints (ochronotic arthropathy).


epilepsy, idiopathic generalized, susceptibility to, 1


Juvenile idiopathic arthritis, extended oligoarthritis


psoriasis-related juvenile idiopathic arthritis

Childhood arthritis typically associated with psoriasis.


Polyarticular Juvenile Idiopathic Arthritis, Rheumatoid Factor Negative

A category of juvenile idiopathic arthritis defined by the presence of arthritis affecting five or more separate joints during the first six months of disease, with negative serologic testing for rheumatoid factor.


FYTIC ACID

Complexing agent for removal of traces of heavy metal ions. It acts also as a hypocalcemic agent.

A myo-inositol hexakisphosphate in which each hydroxy group of myo-inositol is monophosphorylated.


lymphoplasmacytic lymphoma

A clonal neoplasm of small B-lymphocytes, lymphoplasmacytoid cells, and plasma cells involving the bone marrow, lymph nodes, and the spleen. The majority of patients have a serum IgM paraprotein.


pancreatic neoplasm

A benign or malignant neoplasm involving the pancreas.


vulva cancer

A primary or metastatic malignant neoplasm involving the vulva.


Metastatic Biliary Tract Carcinoma

A biliary tract carcinoma that has spread from the original site of growth to other anatomic sites.


EC 1.4.3.4 (monoamine oxidase) inhibitor

An EC 1.4.3.* (oxidoreductase acting on donor CH-NH2 group, oxygen as acceptor) inhibitor that interferes with the action of monoamine oxidase (EC 1.4.3.4).


myotonic syndrome

An inherited or acquired, localized or generalized disorder affecting the muscles. It may be associated with abnormalities in the chloride or sodium channels of the muscles. It is characterized by delayed muscle relaxation following stimulation or contraction. Representative examples include myotonia congenita and myotonic dystrophy.


neonatal diabetes mellitus

Neonatal diabetes mellitus presents as hyperglycemia, failure to thrive and, in some cases, dehydration and ketoacidosis which may be severe with coma, in a child within the first months of life.


otitis media

Inflammation of the anatomical structures of the middle ear, which is most often caused by an infectious process. Symptoms include erythema and edema of the tympanic membrane, pain, and possibly fever.


Substance induction of cytochrome p450 enzyme


plexiform neurofibroma

An elongated and multinodular neurofibroma, formed when the tumor involves either multiple trunks of a plexus or multiple fascicles of a large nerve, such as the sciatic. Some plexiform neurofibromas resemble a bag of worms, others produce a massive ropy enlargement of the nerve. (Adapted from WHO.)


polyarticular juvenile idiopathic arthritis


Localization of sentinel lymph node

biolink:Activity biolink:NamedThing biolink:ActivityAndBehavior biolink:Occurrent biolink:PhysicalEssenceOrOccurrent


vasculitis due to ADA2 deficiency

Vasculitis due to ADA2 deficiency is a rare, genetic, systemic and rheumatologic disease due to adenosine deaminase-2 inactivating mutations, combining variable features of autoinflammation, vasculitis, and a mild immunodeficiency. Variable clinical presentation includes chronic or recurrent systemic inflammation with fever, livedo reticularis or racemosa, early-onset ischemic or hemorrhagic strokes, peripheral neuropathy, abdominal pain, hepatosplenomegaly, portal hypertension, cutaneous polyarteritis nodosa, variable cytopenia and immunoglobulin deficiency.


Abdominal adhesions

Fibrous bands (i.e., bands of scar-like tissue) that span two or more intra-abdominal organs and/or the inner abdominal wall (i.e. peritoneal membrane).


Sodium Chloride

A ubiquitous sodium salt that is commonly used to season food.

An inorganic chloride salt having sodium(1+) as the counterion.


Metamizole

A drug that has analgesic, anti-inflammatory, and antipyretic properties. It is the sodium sulfonate of AMINOPYRINE.

A pyrazole that is antiipyrine substituted at C-4 by a methyl(sulfomethyl)amino group, the sodium salt of which, metamizole sodium, was widely used as a powerful analgesic and antipyretic, but withdrawn from many markets from the 1970s due to a risk of causing risk of causing agranulocytosis.


Manganese Sulfate

A hydrate that is the monohydrate form of manganese(II) sulfate.


Dexamethasone phosphate

A steroid phosphate that is the 21-O-phospho derivative of dexamethasone.


Oxygen

An element with atomic symbol O, atomic number 8, and atomic weight [15.99903; 15.99977]. It is the most abundant element on earth and essential for respiration.


ATROPINE

An alkaloid, originally from Atropa belladonna, but found in other plants, mainly SOLANACEAE. Hyoscyamine is the 3(S)-endo isomer of atropine.

A  racemate composed of equimolar concentrations of (S)- and (R)-atropine . It is obtained from deadly nightshade (Atropa belladonna) and other plants of the family Solanaceae.


Estriol

A hydroxylated metabolite of ESTRADIOL or ESTRONE that has a hydroxyl group at C3, 16-alpha, and 17-beta position. Estriol is a major urinary estrogen. During PREGNANCY, a large amount of estriol is produced by the PLACENTA. Isomers with inversion of the hydroxyl group or groups are called epiestriol.

A 3-hydroxy steroid that is estra-1,3,5(10)-trien-3-ol substituted by additional hydroxy groups at positions 16 and 17 (16alpha,17beta-stereoisomer).


Phthalylsulfathiazole

minor descriptor (63-86); on-line & INDEX MEDICUS search SULFATHIAZOLES (66-86); RN given refers to parent cpd

A sulfonamide incorporating 2-carboxybenzamido and 1,3-thiazol-2-yl moieties that is a broad-spectrum antibiotic indicated in the treatment of dysentery, colitis, gastroenteritis and intestinal surgery.


Abdominal distention

Distention of the abdomen.


ICODEXTRIN

A glucan that is structurally related to maltodextrin, with more than 85% of its molecules having molecular weights between 1640 and 45 000 Daltons (Da), and a weight-average molecular weight of about 20 000 Da; it is used in dialysis fluids as an alternative to glucose-based solutions, and to reduce adhesions after gynecological or abdominal surgery. It has also been used as a vehicle for drugs given via the peritoneal cavity.


Avobenzone

causes allergic or photoallergic contact dermatitis; structure given in first source


Sucrose

A nonreducing disaccharide composed of GLUCOSE and FRUCTOSE linked via their anomeric carbons. It is obtained commercially from SUGARCANE, sugar beet (BETA VULGARIS), and other plants and used extensively as a food and a sweetener.

A glycosyl glycoside formed by glucose and fructose units joined by an acetal oxygen bridge from hemiacetal of glucose to the hemiketal of the fructose.


Fenoterol

A synthetic adrenergic beta-2 agonist that is used as a bronchodilator and tocolytic.

A member of the class  resorcinols that is 5-(1-hydroxyethyl)benzene-1,3-diol in which one of the methyl hydrogens is replaced by a 1-(4-hydroxyphenyl)propan-2-amino group. A beta2-adrenergic agonist, it is used (as the hydrobromide salt) as a bronchodilator in the management of reversible airway obstruction.


Bismuth sub nitrate

antacid containing bismuth, sodium & magnesium carbonates, rutin, Khellin & Rhamnus bark powder; do not confuse with colloidal bismuth subcitrate


Abdominal mass

An abnormal enlargement or swelling in the abdomen.


Titanium Dioxide

used medically as protectant against externally caused irritation & sunlight; high concentrations of dust may cause irritation to respiratory tract; RN given refers to titanium oxide (TiO2); structure

A titanium oxide with the formula TiO2.  A naturally occurring oxide sourced from ilmenite, rutile and anatase, it has a wide range of applications.


Trimebutine

Proposed spasmolytic with possible local anesthetic action used in gastrointestinal disorders.


Nimesulide

An aromatic ether having phenyl and 2-methylsulfonamido-5-nitrophenyl as the two aryl groups.


AMDINOCILLIN PIVOXIL

Pivaloyloxymethyl ester of amdinocillin that is well absorbed orally, but broken down to amdinocillin in the intestinal mucosa. It is active against gram-negative organisms and used as for amdinocillin.


Phloroglucinol

A trinitrobenzene derivative with antispasmodic properties that is used primarily as a laboratory reagent.

A benzenetriol with hydroxy groups at position 1, 3 and 5.


Drotaverine

Hungarian drug; RN given refers to parent cpd; structure


Abdominal rigidity

Involuntary tightening of the abdominal musculature that occurs in response to touching the abdomen to avoid pain. Rigidity can occur in the presence of abdominal inflammation and usually involves only the inflamed area.


Unspecified abortion incomplete

The uterus is not entirely emptied of products of conception following spontaneous, medical or operative pregnancy termination.


Missed abortion

Retention in uterus of an abortus.


Spontaneous abortion

Fetal loss at less than 20 weeks of gestation.


abscess

An inflammatory process characterized by the accumulation of pus within a newly formed tissue cavity which is the result of a bacterial, fungal, or parasitic infection or the presence of a foreign body.


Sterile abscess

An abscess not caused by infection with pyogenic bacteria. Operationally, a sterile abscess is inferred if investigations of an abscess fail to reveal evidence of pathogenic organisms.


Epidermal acanthosis

Diffuse hypertrophy or thickening of the stratum spinosum of the epidermis (prickle cell layer of the skin).


Accident while engaged in work-related activity


Nordazepam

An intermediate in the metabolism of DIAZEPAM to OXAZEPAM. It may have actions similar to those of diazepam.

A 1,4-benzodiazepinone having phenyl and chloro substituents at positions 5 and 7 respectively; it has anticonvulsant, anxiolytic, muscle relaxant and sedative properties but is used primarily in the treatment of anxiety.


eye accommodation disease

Disease that disrupts the process by which the vertebrate eye changes optical power to maintain a clear image or focus on an object as its distance varies.


Fracture of acetabulum

A traumatic break in the portion of the pelvic bone that forms the hip socket.


Flucloxacillin

A penicillin compound having a 6beta-[3-(2-chloro-6-fluorophenyl)-5-methyl-1,2-oxazole-4-carboxamido] side-chain.


Cystic acne

A severe type of acne characterized by the formation of cysts enclosing a mixture of keratin and sebum.


AIDS

A syndrome resulting from the acquired deficiency of cellular immunity caused by the human immunodeficiency virus (HIV). It is characterized by the reduction of the Helper T-lymphocytes in the peripheral blood and the lymph nodes. Symptoms include generalized lymphadenopathy, fever, weight loss, and chronic diarrhea. Patients with AIDS are especially susceptible to opportunistic infections (usually pneumocystis carinii pneumonia, cytomegalovirus (CMV) infections, tuberculosis, candida infections, and cryptococcosis), and the development of malignant neoplasms (usually non-Hodgkin lymphoma and Kaposi sarcoma). The human immunodeficiency virus is transmitted through sexual contact, sharing of contaminated needles, or transfusion of contaminated blood.


skin tag

A small, benign growth that arises from the skin. It is characterized by the presence of fibrovascular tissue lined by epidermis. It may be sessile or pendulous and usually occurs in sites where there is friction.


Activated partial thromboplastin time abnormal


Prolonged partial thromboplastin time

Increased time to coagulation in the partial thromboplastin time (PTT) test, a measure of the intrinsic and common coagulation pathways. Phospholipid, and activator, and calcium are mixed into an anticoagulated plasma sample, and the time is measured until a thrombus forms.


Digitoxin

A cardiac glycoside sometimes used in place of DIGOXIN. It has a longer half-life than digoxin; toxic effects, which are similar to those of digoxin, are longer lasting. (From Martindale, The Extra Pharmacopoeia, 30th ed, p665)

A cardenolide glycoside in which the 3beta-hydroxy group of digitoxigenin carries a 2,6-dideoxy-beta-D-ribo-hexopyranosyl-(1->4)-2,6-dideoxy-beta-D-ribo-hexopyranosyl-(1->4)-2,6-dideoxy-beta-D-ribo-hexopyranosyl trisaccharide chain.


Activated partial thromboplastin time shortened


Acute abdomen

A sudden onset of abdominal pain with associated nausea or vomiting. The acute abdomen may be caused by an infection, inflammation, vascular occlusion, or obstruction.


sweet syndrome

Sweet's syndrome (the eponym for acute febrile neutrophilic dermatosis) is characterized by a constellation of clinical symptoms, physical features, and pathologic findings which include fever, neutrophilia, tender erythematous skin lesions (papules, nodules, and plaques), and a diffuse infiltrate consisting predominantly of mature neutrophils that are typically located in the upper dermis.


Bromazepam

One of the BENZODIAZEPINES that is used in the treatment of ANXIETY DISORDERS.


Parageusia

A distortion of the sense of taste, often characterized by the sensation of a metallic taste.


Acute sinusitis

An acute form of sinusitis.


adenocarcinoma

A common cancer characterized by the presence of malignant glandular cells. Morphologically, adenocarcinomas are classified according to the growth pattern (e.g., papillary, alveolar) or according to the secreting product (e.g., mucinous, serous). Representative examples of adenocarcinoma are ductal and lobular breast carcinoma, lung adenocarcinoma, renal cell carcinoma, hepatocellular carcinoma (hepatoma), colon adenocarcinoma, and prostate adenocarcinoma.


colon adenocarcinoma

A carcinoma that arises from glandular epithelial cells of the colon


adenoid hypertrophy

An upper respiratory tract disease characterized by the unusual growth of the adenoid tonsil; has symptom snoring, has symptom hyponasality, has symptom otitis media with effusion, has symptom mouth breathing.


adenoma

A neoplasm arising from the epithelium. It may be encapsulated or non-encapsulated but non-invasive. The neoplastic epithelial cells may or may not display cellular atypia or dysplasia. In the gastrointestinal tract, when dysplasia becomes severe it is sometimes called carcinoma in situ. Representative examples are pituitary gland adenoma, follicular adenoma of the thyroid gland, and adenomas (or adenomatous polyps) of the gastrointestinal tract.


adrenal cortex adenoma

A benign neoplasm that can arise from any of the adrenal cortical layers. It can be associated with the overproduction of glucocorticoids (Cushing's syndrome), androgenic or estrogenic steroids (adrenogenital syndrome), or mineralocorticoids (Conn's syndrome). (Sternberg Diagnostic Surgical Pathology, 3rd ed.)


adrenal gland disorder

A disease involving the adrenal gland.


Adrenal hemorrhage

Hemorrhage and necrosis of the adrenal gland tissue. Bilateral and extensive hemorrhage may lead to acute adrenal insufficiency, shock, and death.


mood disorder

A cognitive disorder a disturbance in which the person's mood is hypothesized to be the main underlying feature.


Tiapride

A benzamide derivative that is used as a dopamine antagonist.


Lormetazepam

A 1,4-benzodiazepinone compound having a methyl substituent at the 1-position, a hydroxy substituent at the 3-position, a 2-chlorophenyl group at the 5-position and a chloro substituent at the 7-position.


Nitrous Oxide

Nitrogen oxide (N2O). A colorless, odorless gas that is used as an anesthetic and analgesic. High concentrations cause a narcotic effect and may replace oxygen, causing death by asphyxia. It is also used as a food aerosol in the preparation of whipping cream.

A nitrogen oxide consisting of linear unsymmetrical molecules with formula N2O. While it is the most used gaseous anaesthetic in the world, its major commercial use, due to its solubility under pressure in vegetable fats combined with its non-toxicity in low concentrations, is as an aerosol spray propellant and aerating agent for canisters of 'whipped' cream.


Nefopam

Non-narcotic analgesic chemically similar to ORPHENADRINE. Its mechanism of action is unclear. It is used for the relief of acute and chronic pain. (From Martindale, The Extra Pharmacopoeia, 30th ed, p26)

A racemate comprising equal amounts of (R)- and (S)-nefopam. The  hydrochloride is a centrally acting non-opiate analgesic commonly used for the treatment of moderate to severe pain.


Air Embolism

The presence of bubbles of air in the vascular system; occurrence is related to the entry of air into the venous circulation following trauma or surgery.


Carbon Dioxide

A solid form of carbon dioxide used as a refrigerant.

A one-carbon compound with formula CO2 in which the carbon is attached to each oxygen atom by a double bond. A colourless, odourless gas under normal conditions, it is produced during respiration by all animals, fungi and microorganisms that depend directly or indirectly on living or decaying plants for food.


Akinesia

Inability to initiate changes in activity or movement and to perform ordinary volitional movements rapidly and easily.


Elevated circulating alanine aminotransferase concentration

An abnormally high concentration in the circulation of alanine aminotransferase (ALT).


alcohol abuse

The use of alcoholic beverages to excess, either on individual occasions ("binge drinking") or as a regular practice.


Mosapride

Mosapride is a 5-HT4 receptor agonist which increases the release of acetylcholine and stimulates GI motility.

A benzamide resulting from the formal condensation of the carboxy group of 4-amino-5-chloro-2-ethoxybenzoic acid with the amino group of 1-[4-(4-fluorobenzyl)morpholin-2-yl]methanamine.


; RN given refers to isomeric cpd without isomeric designation; mixture of (5E,9E,13E) & (5Z,9E,13E)-isomers

A terpene ketone in which a (9E,13E)-geranylgernayl group is bonded to one of the alpha-methyls of acetone (it is a mixture of 5E- and 5Z-geoisomers in a 3:2 ratio).


Glutathione

A tripeptide with many roles in cells. It conjugates to drugs to make them more soluble for excretion, is a cofactor for some enzymes, is involved in protein disulfide bond rearrangement and reduces peroxides.

A tripeptide compound consisting of glutamic acid attached via its side chain to the N-terminus of cysteinylglycine.


Protionamide

Antitubercular agent similar in action and side effects to ETHIONAMIDE. It is used mostly in combination with other agents.


Albuminuria

Increased concentration of albumin in the urine.


Alcohol Intolerance

Increased sensitivity to the adverse effects of alcohol, which can include nasal congestion, skin flushes, heart dysrhythmias, nausea, vomiting, indigestion and headaches.


Animal protein allergy

Hypersensitivity in form of an adverse immune reaction against animal proteins.


Hydrocortisone butyrate

Cortisol esterified with butyric acid at the 17-hydroxy group.


Elevated circulating alpha-fetoprotein concentration

Concentration of alpha-fetoprotein in the blood circulation above the upper limit of normal.


Flunitrazepam

A benzodiazepine with pharmacologic actions similar to those of DIAZEPAM that can cause ANTEROGRADE AMNESIA. Some reports indicate that it is used as a date rape drug and suggest that it may precipitate violent behavior. The United States Government has banned the importation of this drug.

A 1,4-benzodiazepinone that is nitrazepam substituted by a methyl group at position 1 and by a fluoro group at position 2'. It is a potent hypnotic, sedative, and amnestic drug used to treat chronic insomnia.


Pipamperone

A member of the class of  bipiperidines that is 1,4'-bipiperidine which is substituted at the 1' and 4' positions by 4-(p-fluorophenyl)-4-oxobutyl and carboxamide groups, respectively. A first generation antipsychotic, its properties are generally similar to those of haloperidol.


pulmonary alveolar proteinosis

A rare lung disorder characterized by the filling of the pulmonary alveoli with proteinaceous material which stains positive with periodic acid-Schiff stain. It may be idiopathic or secondary due to hematologic malignancies or the inhalation of mineral dusts. Signs and symptoms include dyspnea, cough and low grade fever.


amaurosis fugax

Transient complete or partial monocular blindness due to retinal ischemia. This may be caused by emboli from the CAROTID ARTERY (usually in association with CAROTID STENOSIS) and other locations that enter the central RETINAL ARTERY. (From Adams et al., Principles of Neurology, 6th ed, p245)


renal aminoaciduria

A group of inherited kidney disorders characterized by the abnormally elevated levels of amino acids in urine. Genetic mutations of transport proteins result in the defective reabsorption of free amino acids at the proximal renal tubules. Renal aminoaciduria are classified by the specific amino acid or acids involved.


Etoricoxib

A sulfone and pyridine derivative that acts as a cyclooxygenase-2 inhibitor. It is used as a NSAID for the treatment of pain associated with RHEUMATOID ARTHRITIS and ANKYLOSING SPONDYLITIS. It is also used for the short-term treatment of moderate postoperative dental pain.

A member of the class of bipyridines that is 2,3'-bipyridine which is substituted at the 3, 5, and 6' positions by 4-(methylsulfonyl)phenyl, chlorine, and methyl groups, respectively.


Canrenoic acid

A synthetic pregnadiene derivative with anti-aldosterone activity.


Dimethindene

A histamine H1 antagonist. It is used in hypersensitivity reactions, in rhinitis, for pruritus, and in some common cold remedies.


Etamsylate

Benzenesulfonate derivative used as a systemic hemostatic.


TERIZIDONE

minor descriptor (1975-1984); on-line & Index Medicus search OXAZOLES (1975-1984); RN given refers to parent cpd without isomeric designation


Canrenone

A synthetic pregnadiene compound with anti-aldosterone activity.


macrocytic anemia

Anemia that is characterized by increased red blood cell volume.


neonatal anemia

The mildest form of erythroblastosis fetalis in which anemia is the chief manifestation.


anemia due to chronic disorder

Anemia due to a disorder that is persistent or long-standing in nature.


Megaloblastic anemia due to vitamin B>12< deficiency


anal fistula

A pathologic tract that connects an opening in the anal canal to the perianal skin. In the vast majority of cases there is a history of perianal abscess.


fibroepithelial polyp of the anus

A non-neoplastic polypoid lesion that arises from the anal canal or perianal skin. It is composed of dense fibrous stroma and it is covered by squamous epithelium.


Anal stenosis

Abnormal narrowing of the anal opening.