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

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

RGX-202 met the main goal of a Phase 3 DMD trial, and Regenxbio plans to seek accelerated approval for the gene therapy.

Seeing two sons with DMD lose the ability to walk isn't making the third son's disease progression any easier, says columnist Betty Vertin.

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 ...

On May 1, my daughter Jill graduated from the University of Pittsburgh with a master’s degree in public policy and management. She also received the department’s student achievement award and was the ...

Duchenne Parent Project Spain is investing €247,000 in a project to create a new laboratory model to study heart health in ...

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 ...

July 2026 Sat 18 MDA Engage – Hershey, PA July 18 @ 8:30 AM - 5:00 PM Hershey Lodge - Confection Hall 325 University Drive, Hershey, PA, United States Join us for an energizing in person gathering ...

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 ...

Bridgebio is funding the MDA with $100,000 to advance projects aimed at improving multidisciplinary care for people with LGMD ...