Muscular Dystrophy News Today brought you daily coverage of the latest scientific findings, treatment developments, and clinical trials related to muscular dystrophy (MD) throughout 2021. We look forward to reporting more news to patients, family members, and caregivers dealing with MD during 2022. Here are the top 10 most-read articles of…

A Phase 1 trial of SRP-9001, Sarepta Therapeutics‘ investigational gene therapy, is recruiting 3-year-old boys with Duchenne muscular dystrophy (DMD) who are able to walk for a new patient group in California, according to a press release from the Muscular Dystrophy Association. This group, being enrolled at the…

Note: This story was updated Oct. 8, 2021, to clarify that Sarepta did not confirm previously announced plans to enroll 75 boys between the ages of 4 and 7 in the EMBARK trial in its latest announcement. Sarepta Therapeutics and Roche have announced the launch of EMBARK,…

Sarepta Therapeutics‘ investigational gene therapy SRP-9001 showed early signs of biologic efficacy in muscle biopsies from 11 boys with Duchenne muscular dystrophy (DMD) treated in a Phase 1 clinical trial, the company announced in a press release. “Robust” expression of micro-dystrophin was evident in cells at 12…

The clinical program evaluating the safety, tolerability, and efficacy of RG6206 in boys with Duchenne muscular dystrophy (DMD) has been discontinued, according to a letter to the DMD community by Roche and Genentech. “We recognize this news is deeply disappointing for the Duchenne community, especially in…

Climbing up the world’s tallest mountain isn’t for everyone. With that in mind, a small New England-based charity plans just such an adventure this November — for the third time, no less — to raise at least $100,000 to help cure Duchenne muscular dystrophy (DMD). Tonya Dreher is executive…

Back in June 1984, a doctor calmly informed Pat Furlong that her sons — 4-year-old Chris and 6-year-old Patrick — had Duchenne muscular dystrophy. It was the first time she had ever heard those three words uttered together. The disease eventually claimed both boys, but that didn’t stop Furlong’s fight against…

Sarepta Therapeutics is partnering with Roche to accelerate the development and expand the availability of SRP-9001 — its leading gene therapy candidate for Duchenne muscular dystrophy (DMD) — outside the Unites States. DMD is caused by deficient production of dystrophin — an essential protein for…

Emily Crossley was a well-known TV correspondent for Britain’s Channel 4, and a presenter for CNN International’s World Business Today program. But her own world was turned upside-down the day she learned her son, Eli, had Duchenne muscular dystrophy (DMD). “The initial diagnosis was so devastating. There’s no escape. It’s…

Discussions of the work being done on four different approaches to treating Duchenne muscular dystrophy (DMD) — repairing or replacing dystrophin, managing inflammation, improving muscle mechanics, and editing dysfunctional genes using CRISPR/CAS9 — were a highlight of sessions at the recent 2018 Parent Project Muscular Dystrophy (PPMD) conference in Scottsdale, Arizona.