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Zilganersen
go.drugbank.com/drugs/DB18161ApprovedInvestigational[L64096] Alexander disease is caused by variants in the *GFAP* gene that lead to overproduction and toxic accumulation of GFAP in astrocytes, and dysfunction in astrocytes can over time damage neurons … [L64096] The FDA approved zilganersen on September 3, 2026 for the treatment of Alexander disease in pediatric and adult patients, making it the first and only disease-modifying treatment for the condition
Allovectin-7
go.drugbank.com/drugs/DB17439InvestigationalIt was discontinued after it failed to improve the objective response rate or overall survival versus first-line chemotherapy in malignant melanoma.[A255207] … Allovectin-7 or allovectin-7(r) is a lipid complex that contains the DNA sequences encoding HLA-B7 and beta-2 microglobulin.
Danicopan
go.drugbank.com/drugs/DB15401ApprovedInvestigational[A263506] It was subsequently approved by the FDA in March 2024.[L50386] … [L50381] It was first approved in January 2024 in Japan for patients with PNH,[A263506,L50416] shortly after which the EMA adopted a positive opinion and recommended granting it marketing authorization
Synonyms: (2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidin-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridin-2-yl)-4-fluoropyrrolidine-2-carboxamide, (2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidine-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridine-2-yl)-4-fluoropyrrolidine-2-carboxamideTividenofusp alfa
go.drugbank.com/drugs/DB17628ApprovedInvestigationalTividenofusp alfa is a first-in-class, brain-penetrant enzyme replacement therapy (ERT) indicated for the treatment of Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome [L56097, L56099 … Hunter syndrome is a rare, X-linked lysosomal storage disorder caused by a deficiency in the enzyme iduronate-2-sulfatase (IDS), which leads to the toxic accumulation of glycosaminoglycans (GAGs) throughout
Aducanumab
go.drugbank.com/drugs/DB12274ApprovedInvestigationalin amyloid-β plaques. … [L34420] Biogen enrolled patients in phase 3 clinical trials in 2015 but increased the size of the trials from 1350 patients to 1650 patients to maintain statistical power in the face of a high standard
Natural Killer Cell
go.drugbank.com/drugs/DB15721InvestigationalThese cells arise from CD34+ lymphoid progenitors and comprise 10-15% of all lymphocytes in human peripheral blood. … Expansion involves the use of feeder cell line systems or bioreactors to enhance the number of NK cells with desired cytotoxicity.
Voxilaprevir
go.drugbank.com/drugs/DB12026ApprovedInvestigationalHCV is a single-stranded RNA virus that is categorized into nine distinct genotypes, with genotype 1 being the most common in the United States, and affecting 72% of all chronic HCV patients [L852]. … Notably, Vosevi is approved for use in patients with genotypes 1-6 who have been previously treated with an NS5A inhibitor, or patients with genotypes 1a or 3 infection and have previously been treated
Queuine
go.drugbank.com/drugs/DB14732ExperimentalNutraceuticalAll eukaryotic organisms, including humans, transform queuine to queuosine by placing it in the wobble position (anticodon) of several tRNAs including aspartic acid, asparagine, histidine, and tyrosine … Quantities of queuine have been found in tomatoes, wheat, coconut water, and milk from humans, cows, and goats. Humans salvage and recover queuine from either ingested food or the gut flora.
Macitentan
go.drugbank.com/drugs/DB08932ApprovedInvestigationalMacitentan is a dual endothelin receptor antagonist used in the treatment of pulmonary arterial hypertension (PAH).[L35890] It was first approved by the FDA in 2013. … A combination product (Opsynvi) comprising macitentan and [tadalafil] was approved in Canada in October 2021 for the treatment of PAH.[L39105] It was subsequently approved by the FDA in March 2024.
Disulfiram
go.drugbank.com/drugs/DB00822ApprovedInvestigationalA carbamate derivative used as an alcohol deterrent. It is a relatively nontoxic substance when administered alone, but markedly alters the intermediary metabolism of alcohol. … It acts by inhibiting aldehyde dehydrogenase.
Categories: Drugs Used in Alcohol Dependence, Drugs Used in Addictive DisordersSynonyms: N,N,N',N'-tetraethylthiuram disulfide, 1,1'-dithiobis(N,N-diethylthioformamide)