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Eteplirsen
go.drugbank.com/drugs/DB06014ApprovedInvestigationalEteplirsen was the first treatment for DMD approved by the FDA.[A244925] … [L40015] Duchenne muscular dystrophy is a rare genetic disorder characterized by progressive muscle deterioration and premature death most commonly due to respiratory or cardiac complications.
Torasemide
go.drugbank.com/drugs/DB00214ApprovedInvestigational[A319] Torasemide was first approved for clinical use by the FDA in 1993.[L5257]
Elosulfase alfa
go.drugbank.com/drugs/DB09051ApprovedInvestigationalIt was approved by the FDA in 2014 for the treatment of Morquio syndrome. Elosulfase alfa was developed by BioMarin Pharmaceutical Inc. and is marketed under the brand Vimizim™.
Omega-3 fatty acids
go.drugbank.com/drugs/DB11133ApprovedInvestigationalNutraceuticalα-linolenic acid (ALA) (found in plant oils), eicosapentaenoic acid (EPA), and docosahexaenoic acid (DHA) (both commonly found in fish oil that originally come from microalgae that is further consumed by
Melatonin
go.drugbank.com/drugs/DB01065ApprovedInvestigationalNutraceuticalVet approvedIn mammals, melatonin is produced by the pineal gland. … pineal gland is small endocrine gland, about the size of a rice grain and shaped like a pine cone (hence the name), that is located in the center of the brain (rostro-dorsal to the superior colliculus) but
Eptinezumab
go.drugbank.com/drugs/DB14040ApprovedInvestigationalEptinezumab is a fully-humanized IgG1 antibody manufactured using yeast (_Pichia pastoris_) and developed by Lundbeck Seattle Biopharmaceuticals. … [F94,A33105,A33106,A33108] It was approved by the FDA in February 2020 for the preventive treatment of migraine headaches in adults.[L12318]
Aficamten
go.drugbank.com/drugs/DB18490ApprovedInvestigational[L54783] Aficamten works by attenuating myocardial hypercontractility and left ventricular outflow tract obstruction, which are characteristics of oHCM.[A275293] … [L54788] Approved by the FDA on December 19, 2025, aficamten is indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and
Natalizumab
go.drugbank.com/drugs/DB00108ApprovedInvestigational[A261331] On August 24, 2023, the first biosimilar to natalizumab, natalizumab-sztn, was approved by the FDA.[L48041] Natalizumab was approved by the European Commission on September 22, 2023. … [A261326] Natalizumab was further approved by the FDA for the treatment of Crohn's Disease in January 2008.
Cefadroxil
go.drugbank.com/drugs/DB01140ApprovedInvestigationalVet approvedWithdrawnLong-acting, broad-spectrum, water-soluble, cephalexin derivative.
Estradiol
go.drugbank.com/drugs/DB00783ApprovedInvestigationalVet approvedEstradiol is a naturally occurring hormone circulating endogenously in females. It is commercially available in several hormone therapy products for managing conditions associated with reduced estrogen, such as vulvovaginal atrophy and h...