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The Muscular Dystrophy Association celebrates the U.S. Food and Drug Administration’s expanded approval of ELEVIDYS.

The Muscular Dystrophy Association’s funding of foundational research leads to new drug approval by the US Food and Drug Administration (FDA) of Duvyzat (givinostat), a histone deacetylase (HDAC) inhibitor to treat children and adolescents living with Duchenne muscular dystrophy (DMD).

Capricor Therapeutics today announced that the U.S. Food and Drug Administration (FDA) has scheduled a Pre-BLA (Biologics License Application) meeting with the Company in the third quarter of 2024 for deramiocel (CAP-1002), for the treatment of Duchenne Muscular Dystrophy (DMD).

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association, provided updates in its latest Newsletter on results of its gene therapy clinical trials for Duchenne Muscular Dystrophy and advances in gene therapies for limb-girdle muscular dystrophies by its spinoff Atamyo Therapeutics.

Constant Therapeutics LLC today announced that the first patient was dosed March 3, 2024, in the Company’s phase 2 clinical trial of TXA127, the Company’s lead peptide product under development as a potential treatment for Duchenne Muscular Dystrophy-associated (DMD-associated) Cardiomyopathy.

Today, the U.S. Food and Drug Administration expanded the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD) for ambulatory and non-ambulatory individuals 4 years of age and older with DMD with a confirmed mutation in the DMD gene.

Pfizer’s investigational Duchenne muscular dystrophy gene therapy, fordadistrogene movaparvovec, failed in a late-stage study to significantly improve motor function in patients versus placebo.

The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of AGAMREE® (vamorolone), a structurally unique steroidal anti-inflammatory drug to treat children and adolescents living with Duchenne muscular dystrophy (DMD).

Elixirgen Therapeutics, Inc., today presented preclinical data on its proprietary Bobcat mRNA TM technology in a poster presentation at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference taking place March 3-6, 2024, in Orlando, Florida.

Avidity Biosciences, Inc. today announced it is partnering with the FSHD Society and joining the global community of patients, caregivers, and healthcare providers to raise awareness of facioscapulohumeral muscular dystrophy in support of World FSHD Day.