6 天之前 · Phase 1/2 trial of exon 44 skipping therapy for DMD cleared in UK. Entrada Therapeutics announced it has been cleared in the U.K. to start a Phase 1/2 clinical trial of …

2025年1月2日 · 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 …

6 小时之前 · The “impressive results” are a “pivotal moment” for developer Cumberland Pharmaceuticals, “and, more importantly, for the DMD community,” A.J. Kazimi, CEO of …

2 天之前 · 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. ENTR-601 …

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 …

2024年1月2日 · 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 …

2025年1月28日 · 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 …

2025年1月9日 · 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 …

2024年12月17日 · “This landmark study presents compelling biomarker data and promising signals that suggest the potential for functional stabilization with administration of …

2024年8月8日 · 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 …