The FDA has given the green light to a clinical trial testing ATA-200, a gene therapy for limb-girdle muscular dystrophy ...
Despite positive trial data, Sarepta Therapeutics has decided to stop the clinical development of SRP-5051 (vesleteplirsen), ...
Columnist Robin Stemple says he chooses to focus on what FSHD progression hasn't taken from him. The small victories matter, ...
Specific mutations in the DMD gene influence how long patients retain the ability to walk, even when treated with ...
I have three teenage sons — Max, 18, Rowen, 15, and Charlie, 13 — who have Duchenne muscular dystrophy (DMD). They have met several sweet milestones over the years, and I love celebrating them. When ...
Three weeks ago, my girlfriend, Amanda, and I embarked on a unique journey by co-founding Rebirth Ensemble (RE), an art-making business with a distinct purpose. This partnership is deeply rooted in ...
A new patient group is being enrolled in a Phase 1/2 trial of delpacibart braxlosiran (del-brax), an experimental and potentially disease-modifying therapy for facioscapulohumeral muscular dystrophy ...
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