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Viltolarsen
go.drugbank.com/drugs/DB15005ApprovedInvestigationalDuchenne muscular dystrophy (DMD) is an X-linked recessive allelic disorder characterized by a lack of functional dystrophin protein, which leads to progressive ambulatory, pulmonary, and cardiac function … Viltolarsen was developed by Nippon Shinyaku Co LTD and is being marketed under the name VILTEPSO™.[L15526]
Rilzabrutinib
go.drugbank.com/drugs/DB17709ApprovedInvestigational[L53743] ITP is an autoimmune disorder characterized by low platelet count. … Rilzabrutinib is an oral, reversible, covalent inhibitor of Bruton's tyrosine kinase [A259078, L53738] that was approved by the FDA on August 29, 2025, for the treatment of persistent and chronic immune
Categories: P-glycoprotein inhibitors, P-glycoprotein substratesSynonyms: (e/z)-(r)-2-(3-(4-amino-3-(2-fluoro-4-phenoxyphenyl)-1h-pyrazolo(3,4-d)pyrimidin-1-yl)piperidine-1-carbonyl)-4-methyl-4-(4-(oxetan-3-yl)piperazin-1-yl)pent-2-enenitrile, (3R)-3-[4-Amino-3-(2-fluoro-4-phenoxyphenyl)-1H-pyrazolo[3,4-d]pyrimidin-1-yl]-?-[2-methyl-2-[4-(3-oxetanyl)-1-piperazinyl]propylidene]-?-oxo-1-piperidinepropanenitrileIdebenone
go.drugbank.com/drugs/DB09081ApprovedInvestigationalDue to its biochemical mode of action, it's thought that idebenone may re-activate viable-but-inactive retinal ganglion cells (RGCs) in LHON patients [L885]. … It is not currently approved for use by either the Food and Drug Administration (USA) or Health Canada.
Mixtures: EVERON 90/5 MG FILM KAPLI TABLET ,50 TABLET, EVERON 90/5 MG FILM KAPLI TABLET ,100 TABLETCategories: Compounds used in a research, industrial, or household settingOmalizumab
go.drugbank.com/drugs/DB00043ApprovedInvestigationalBy binding to IgE and neutralizing it, omalizumab reduces free IgE levels and prevents IgE from binding to its receptors on immune cells. … [A39518] Omalizumab was first approved in the US in 2003 [A263507] and in Europe in 2005.
Categories: Agents to Treat Airway DiseaseProducts: XOLAIR SOLUTION FOR INJECTION IN PRE-FILLED SYRINGE 150MG/1.0ML, XOLAIR SOLUTION FOR INJECTION IN PRE-FILLED SYRINGE 75MG/0.5MLMidazolam
go.drugbank.com/drugs/DB00683ApprovedIllicitInvestigational[F2977] This drug was initially approved by the US FDA in 1985, and has been approved for various indications since. … and endoscopic procedures as pre-anesthetic medication, and as an adjunct to local anesthesia.
Categories: P-glycoprotein substrates, Cytochrome P-450 SubstratesProducts: N/a, DEMIZOLAM 5 MG/5 ML IV/IM ENJEKTABL SOLUSYON ICEREN AMPUL, 5 ADETLormetazepam
go.drugbank.com/drugs/DB13872ApprovedLormatazepam is an orally available benzodiazepine used in the UK for the treatment of short-term insomnia [L927]. … It is marketed by Auden Mckenzie (Pharma Division) in 0.5 and 1 mg tablet formulations.
Categories: Heterocyclic Compounds, 2-RingSynonyms: N-Methyllorazepam, 7-Chloro-1,3-dihydro-5-(o-chlorophenyl)-3-hydroxy-1-methyl-2H-1,4-benzodiazepin-2-oneCorticosterone Methyl Oxidase I Deficiency (CMO I)
smpdb.ca/view/SMP0000577DiseaseCorticosterone methyloxidase type I (CMO-I) deficiency, also known as 18-hydroxylase deficiency or aldosterone deficiency among other names, is a genetic disorder that is autosomally linked and caused … In CMO-I deficiency, aldosterone levels are so low that they are undetectable in plasma.
Sepiapterin
go.drugbank.com/drugs/DB16326ApprovedInvestigational[L53548,L53593] Sepiapterin was granted marketing authorization by the European Commission in June 2025 and by the US FDA in July 2025 for the treatment of adults and children with phenylketonuria. … It is a natural precursor of the enzymatic co-factor tetrahydrobiopterin (BH4), which activates PAH and helps reduce blood phenylalanine levels by enhancing the conformational stability of misfolded PAH
Categories: Heterocyclic Compounds, 2-RingSynonyms: L-sepiapterinIL15-NK Cell
go.drugbank.com/drugs/DB15725InvestigationalNatural killer (NK) cells can be incubated with ‘feeder cells’ expressing certain cytokines prior to maturation to enhance their activity. … In particular, incubation with IL-15 allows NK cells to lyse a broad range of fresh and cultured tumor targets that the cell is not normally sensitive to.
Synonyms: IL15-NK CellLeucine
go.drugbank.com/drugs/DB00149ApprovedInvestigationalNutraceuticalAn essential branched-chain amino acid important for hemoglobin formation.
Salts: L-Leucine hydrochlorideMixtures: TRAVASOL SOLUCION 8.5% CON ELECTROLITOS, PF K-CAMINE %8 AMINO ASIT IV INFUZYON ICIN COZELTI, 1000 ML (SETLI)