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Etranacogene 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.
Categories: Complex MixturesZilganersen
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
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-carboxamideAcetic acid
go.drugbank.com/drugs/DB03166ApprovedInvestigational(Stedman, 26th ed) Acetic acid otic (for the ear) is an antibiotic that treats infections caused by bacteria or fungus.
Mixtures: AMINOSTERIL N-HEPA 8%, AMINOSTERIL N-HEPA 500 ML SOLUSYONCategories: Compounds used in a research, industrial, or household settingTurpentine
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, Complex MixturesRisperidone
go.drugbank.com/drugs/DB00734ApprovedInvestigational[L12885] Schizophrenia and various mood disorders are thought to be caused by an excess of dopaminergic D2 and serotonergic 5-HT2A activity, resulting in overactivity of central mesolimbic pathways … Risperidone is thought to reduce this overactivity through inhibition of dopaminergic D2 receptors and serotonergic 5-HT2A receptors in the brain.
Asciminib
go.drugbank.com/drugs/DB12597ApprovedInvestigational[L39005] It has also shown benefit in Ph+ CML with the T315I mutation, which produces a mutant BCR-ABL1 which is typically treatment-resistant as compared to wild-type BCR-ABL1. … [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.
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.
Categories: Complex MixturesInebilizumab
go.drugbank.com/drugs/DB12530ApprovedInvestigational[L52705] In December 2025, the FDA approved an additional indication for generalized myasthenia gravis. [L54838] … Compared to the anti-CD20 antibody [rituximab], which is also used to treat NMOSD, inebilizumab has broader specificity.
Eflornithine
go.drugbank.com/drugs/DB06243ApprovedInvestigationalWithdrawn[A262823,L49318] Subsequently, on December 14, 2023, the FDA approved eflornithine again but under the brand name IWILFIN as an oral maintenance therapy to reduce the risk of relapse in adult and pediatric … a 68% reduction in the risk of death were observed.