News

Rich Horgan’s younger brother, Terry Horgan, is the inspiration behind Cure Rare Disease, a Boston-based nonprofit Rich established in 2017 to help scientists develop personalized therapies for people with rare diseases. Terry, 24, has Duchenne muscular dystrophy (DMD), which is the organization’s first focus. Besides Terry, three other…

Duchenne UK is partnering with Catabasis Pharmaceuticals to provide financial support for a Phase 2 clinical trial testing the experimental oral therapy edasalonexent in non-ambulatory boys and men with Duchenne muscular dystrophy (DMD). The charity is granting more than $600,000…

Montana, the fourth-largest U.S. state in area, also is one of the least-populated — with maybe half a dozen Duchenne muscular dystrophy patients among its 1.1 million inhabitants. One of them is 5-year-old Grant Heaton, the son of cattle ranchers Paul and Laura Heaton. Diagnosed with Duchenne at age…

Solid Biosciences announced it will reduce its workforce by approximately one third to cut expenses and focus on advancing the development of SGT-001, its gene therapy candidate for the treatment of Duchenne muscular dystrophy (DMD). SGT-001 uses a viral vector to deliver a…

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…