4 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) …

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 …

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 …

6 days ago · 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 …

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 …

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 4, 2022 · In May, Pfizer announced a delay in the start of its Phase 3 clinical trial — called CIFFREO (NCT04281485) — in the U.S.The global trial, to be held at 29 locations, was …

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 …

Jan 5, 2021 · Throughout 2020, Muscular Dystrophy News Today brought you daily coverage of important discoveries, treatment advancements, clinical trial findings, and other relevant …