Dystrophy

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…

VANCOUVER, British Columbia, July 11, 2023 (GLOBE NEWSWIRE) -- SOLVE FSHD, a venture philanthropy organization catalyzing innovation and removing barriers to accelerate new therapies for FSHD, announced today that it has awarded grants…

New York, June 27, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of RYSTIGGO (rozanolixizumab-noli) for the treatment of generalized myasthenia gravis…

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 20, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of subcutaneous (SC) Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc),for the…

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…

BOSTON, June 13, 2023 (GLOBE NEWSWIRE) -- PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular…

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…

BOSTON, May 30, 2023 (GLOBE NEWSWIRE) -- PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular…

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…