5 days ago · MDA honors researcher, patient advocate with 2025 Legacy Awards. The Muscular Dystrophy Association (MDA) has announced its 2025 Legacy Awards, and will honor two …

Jan 2, 2025 · Read the story here. No. 8 – PPMD conference highlighted DMD gene therapy and treatments. As part of our coverage of the 2024 Parent Project Muscular Dystrophy (PPMD) …

22 hours ago · The goal of exon-skipping therapy is to allow the body to make a shorter but functional form of the dystrophin protein by skipping over a specific exon on the DMD gene. …

FDA awards fast track designation to DM1 treatment DYNE-101. The U.S. Food and Drug Administration (FDA) has granted fast track designation to DYNE-101, a treatment candidate …

Jan 2, 2024 · No. 10 – With EMBARK trial data, Sarepta seeks to expand Elevidys’ approval. Elevidys (delandistrogene moxeparvovec-roki) is a one-time gene therapy approved in the …

Jan 28, 2025 · That’s according to top-line findings from part two of the Phase 3 EMBARK trial (NCT05096221), which evaluated Sarepta Therapeutics‘ approved gene therapy in more than …

Dec 19, 2024 · DMD is caused by gene mutations that lead to a lack of dystrophin — a protein that normally acts like a shock absorber in muscle cells to limit damage during muscle …

Jan 9, 2025 · Gene-editing therapy targets DYSF, whose mutations cause LGMD type 2B/R2. MD refers to a group of disorders characterized by progressively worsening muscle weakness and …

Aug 8, 2024 · Scientific advances have helped those with DMD live longer and richer lives. A number of treatments can help with symptoms and even slow disease progression including …

Jul 5, 2024 · Sarepta’s Elevidys is 1st FDA-approved DMD gene therapy. A few weeks ago, Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec-rokl) became the first gene …