A final decision from the FDA is expected by Nov. 27, and if approved, BBP-418 would become the first FDA-approved therapy ...

Dyne launched a global clinical trial, FORZETTO, to test its exon-skipping therapy z-rostudirsen in boys with Duchenne ...

Columnist Shalom Lim is grateful for the opportunity to use his lived experience to do good work on behalf of people with DMD ...

Having a rare disease is not an easy life. I have limb-girdle muscular dystrophy, so in addition to the physical decline of my body, I also need to be hyperaware of my mental health. I don’t think ...

The Phase 3 portion of a clinical trial testing RGX-202, an investigational gene therapy for Duchenne muscular dystrophy (DMD), met its main goal, the therapy’s developer, Regenxbio, announced in a ...

ATA-200, an experimental gene therapy for children with LGMDR5, has so far been tolerated well in an early clinical trial.

September 2026 Thu 17 Sam Osborn Memorial Golf Tournament September 17 - September 18 Tiburon Golf Club & Banquet Facility 10302 SOUTH 168TH STREET, OMAHA, NE, United States Join us for the annual Sam ...

MDA Engage – Chicago September 25 @ 7:00 PM - September 26 @ 4:45 PM Westin Chicago Lombard 70 Yorktown Shopping Center, Lombard, IL, United States Events Today Next Events Subscribe to calendar ...

Join us for a day of progress! Let’s unite our communities and bring awareness to neuromuscular disease. Together with MDA, we will continue to lead the way in accelerating care, […] Previous Events ...

This article was provided by our partner, the Muscular Dystrophy Association. It has been reviewed by Bionews for accuracy and relevance. The views and opinions expressed are those of the author and ...

Join us for the annual Sam Osborn Memorial Golf Tournament, where we come together on the fairways to celebrate the life of an extraordinary individual and raise funds for causes close to Sam's heart.