Duchenne

CORAL GABLES, Fla., Nov. 02, 2023 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing, developing, and commercializing novel…

Vamorolone is a Promising Best-In-Class Dissociative Anti-Inflammatory Steroid Treatment for Duchenne Muscular Dystrophy Synergistic Novel Asset Leverages Catalyst's Expertise and Bolsters Catalyst's Rare Neuroscience Portfolio…

New York, June 22, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatment of…

New York, June 15, 2023 (GLOBE NEWSWIRE) -- June 15, 2023 -- CureDuchenne, Muscular Dystrophy Association (MDA), and Parent Project Muscular Dystrophy (PPMD), today announced a collaborative clinical trial grant to test repurposing of…

SAN DIEGO, June 13, 2023 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and…

FDA grants orphan drug designation for MetrioPharm’s lead compound MP1032 for the treatment of Duchenne muscular dystrophy (DMD)DMD is a designated orphan disease in both Europe and the U.S.MetrioPharm’s development objectives: better…

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Breadcrumb Trail Links PMN Business Author of the article: Published May 12, 2023  •  2 minute read Article content Advisers to the U.S. Food and Drug Administration are meeting on Friday to review Sarepta…

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Mutations of the gene encoding dystrophins have long been known to cause the debilitating muscle-wasting disease DMD, which affects one in every 5,000 boys born. People with the condition will usually only live into their 20s or 30s. Now, a…