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Paraldehyde
go.drugbank.com/drugs/DB09117ApprovedWithdrawnParaldehyde is used as an ingredient in some cough medicines as an expectorant, but its efficacy for this indication has not been confirmed and its use as an expectorant may possibly be due to a placebo … Paraldehyde was initially introduced into medical practice in the United Kingdom in 1882 by the Italian physician Vincenzo Cervello.
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
Delandistrogene moxeparvovec
go.drugbank.com/drugs/DB16802ApprovedInvestigational[L47026] DMD is an X-linked genetic disorder characterized by mutations in the dystrophin gene, leading to a deficiency in functional dystrophin protein. … [A260256] DMD tends to be more prevalent in males.
Sonidegib
go.drugbank.com/drugs/DB09143ApprovedInvestigationalSonidegib is a Hedgehog signaling pathway inhibitor (via smoothened antagonism) developed as an anticancer agent by Novartis. It was FDA approved in 2015 for the treatment of basal cell carcinoma.
Synonyms: N-[6-(cis-2,6-dimethylmorpholin-4-yl)pyridin-3-yl]-2-methyl-4'-(trifluoromethoxy)[1,1'-biphenyl]-3-carboxamideFerric cation
go.drugbank.com/drugs/DB13949ApprovedWithdrawnIron is an essential element involved in various metabolic processes, including oxygen transport, deoxyribonucleic acid (DNA) synthesis, and energy production in electron transport [A32514]. … By mass, it is the most common element on Earth.
Moxisylyte
go.drugbank.com/drugs/DB09205ApprovedMoxisylyte, denominated as thymoxamine in the UK, is a specific and orally active α1-adrenergic antagonist. … [L1172] This drug was developed by the Japanese company Fujirebio and also by the American company Iolab in the late 80s.
Categories: Drugs Used in Erectile DysfunctionEtranacogene dezaparvovec
go.drugbank.com/drugs/DB16791ApprovedInvestigationalHemophilia B - also called factor IX deficiency or Christmas disease - is an X-linked genetic disorder resulting in an absence or deficiency of clotting factor IX. … [L44156] It delivers a copy of the deficient gene that results in cell transduction and an eventual increase in circulating factor IX activity.
Asciminib
go.drugbank.com/drugs/DB12597ApprovedInvestigational[A241065] Asciminib is unique in that it acts as an allosteric inhibitor, binding at the myristoyl pocket of the BCR-ABL1 protein and locking it into an inactive conformation. … More specifically, it is an inhibitor of the ABL1 kinase activity of the BCR-ABL1 fusion protein[L38995] which serves as a driver of CML proliferation in most patients with the disease.
Asparaginase Erwinia chrysanthemi
go.drugbank.com/drugs/DB08886ApprovedInvestigationalAsparaginase _Erwinia chrysanthemi_ is an asparaginase-specific enzyme derived from _Erwinia_ _chrysanthemi_ used as an anticancer agent. … It works by depleting the stores of an important amino acid called asparagine, which is involved in DNA synthesis and cell survival of malignant cells, leading to cell death.
Turpentine
go.drugbank.com/drugs/DB11120ApprovedIt is used as a solvent and base material in organic synthesis reactions.
Categories: Compounds used in a research, industrial, or household setting