Tonya Dreher wanted to do something dramatic on behalf of Hope for Gus, the New Hampshire-based foundation inspired by her son, Gus, 14, who has Duchenne muscular dystrophy (DMD). In 2015, frustrated with her fundraising efforts on behalf of DMD research, she came up with the idea of…
Parent Project Muscular Dystrophy (PPMD) has granted $465,000 to Renzhi Han, PhD, a professor at the Ohio State University College of Medicine, to advance research into a targeted gene editing approach that may protect cardiac and skeletal muscle function in people with Duchenne muscular dystrophy (DMD).
Astellas Pharma recently agreed to acquire Audentes Therapeutics, a move it expects will result in faster development of potentially best-in-class therapies for rare neuromuscular diseases, including muscular dystrophy (MD). Audentes’ vectorized exon-skipping technology — which uses a modified adeno-associated virus (AAV) vector to allow cells to…
Throughout 2019, Muscular Dystrophy News Today brought you daily coverage of important discoveries, treatment developments, clinical trials, and other important events related to muscular dystrophy. As we look forward to bringing you more news this year, we present the 10 most-read stories of 2019. No. 10 – “Sarepta…
The newly approved therapy Vyondys 53 (golodirsen) is available for immediate distribution to treat people with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, according to the medicine’s developer Sarepta Therapeutics. In an email reply to Muscular Dystrophy News Today, a spokeswoman added that, similar to Exondys 51 (eteplirsen, also…
Treatment with gene therapy candidate SGT-001 may lead to clinically meaningful improvements for patients with Duchenne muscular dystrophy (DMD), new data from two participants in the Phase 1/2 IGNITE DMD trial suggest. Solid Biosciences, which developed the gene therapy, is currently working with the U.S. Food…
The experimental therapy ATL1102 is safe and continues to show evidence of improved upper muscle strength and function in all nine non-ambulatory boys with Duchenne muscular dystrophy (DMD) in a nearly complete Phase 2 trial in Australia, updated data show. Final results from the open-label study, testing ATL1102…
Following its failure to increase dystrophin levels of patients in an early-stage clinical trial, Wave Life Sciences has discontinued development of the investigational exon-skipping therapy suvodirsen for people with Duchenne muscular dystrophy (DMD). Lack of dystrophin, an essential protein for healthy muscles, causes DMD. Patients may…
The U.S. Food and Drug Administration (FDA) has authorized use of the first test intended to facilitate newborn screening for Duchenne muscular dystrophy (DMD). “Diagnostics that can safely and effectively screen newborns can help health care professionals identify and discuss potential treatment options with parents and caregivers before symptoms…
The U.S. Food and Drug Administration has conditionally approved Vyondys 53 (golodirsen) to treat Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping. This accelerated approval comes less than four months after the FDA issued a complete response letter rejecting Sarepta Therapeutics’ application for…
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