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Olipudase alfa
go.drugbank.com/drugs/DB12835ApprovedInvestigational[A251590] It was later approved by the European Commission on June 28, 2022 [L42740] and by the FDA on August 31, 2022.[L43145] … [L42740] ASMD is a rare lysosomal storage disease caused by mutations in the SMPD1 gene, leading to a deficiency in acid sphingomyelinase and the abnormal accumulation of the primary ASM substrate, sphingomyelin
Elagolix
go.drugbank.com/drugs/DB11979ApprovedInvestigationalunmet needs of women who are affected by endometriosis, depending on their specific type and severity of endometriosis pain. … In particular, estimates suggest that one in ten women of reproductive age is affected by endometriosis and experience debilitating pain symptoms [A35868, A35869, F801].
Abciximab
go.drugbank.com/drugs/DB00054ApprovedWithdrawnAbciximab binds to the glycoprotein (GP) IIb/IIIa receptor of human platelets and inhibits platelet aggregation by preventing the binding of fibrinogen, von Willebrand factor, and other adhesive molecules
Lyme disease vaccine (recombinant OspA)
go.drugbank.com/drugs/DB00045ApprovedWithdrawnVaccine against Lyme disease that contains lipoprotein OspA, an outer surface protein of Borrelia burgdorferi sensu stricto ZS7, as expressed by Escherichia coli.
Thyroid, porcine
go.drugbank.com/drugs/DB09100ApprovedInvestigational[A190831,L11755] Thyroid extract has been described in literature to treat hypothyroidism since 1891[A190807] but its use dates back as far as the 6th century.
Rifapentine
go.drugbank.com/drugs/DB01201ApprovedInvestigationalSpecifically, it interacts with bacterial RNA polymerase but does not inhibit the mammalian enzyme.
Zilganersen
go.drugbank.com/drugs/DB18161ApprovedInvestigational[L64096] It is administered by intrathecal injection, and requires dilution with an accompanying artificial cerebrospinal fluid diluent before administration. … [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
Cystine
go.drugbank.com/drugs/DB00138ApprovedInvestigationalNutraceuticalWithdrawnA covalently linked dimeric nonessential amino acid formed by the oxidation of cysteine. Two molecules of cysteine are joined together by a disulfide bridge to form cystine.
Tetrachlorodecaoxide
go.drugbank.com/drugs/DB05389ApprovedWF10 is a chlorite-based, immunomodulating drug is developed by Nuvo Research Inc. Certain preclinical evidence and clinical pilot data suggest that WF10 may be effective in treating certain cancers. … The Corporation believes the research to-date demonstrates that WF10 acts on macrophages (a type of white blood cell) by modulating the balance between inflammation and phagocytosis, a state in which the
Nelarabine
go.drugbank.com/drugs/DB01280ApprovedInvestigational[A258719] Nelarabine was first granted accelerated approval by the FDA on October 28, 2005, and was manufactured under the trademark name ARRANON by GlaxoSmithKline. … [A15220] Subsequently, nelarabine was also approved by both Health Canada and European Medicines Agency in 2007 under the trademark name ATRIANCE.[L45874,L45879]