Luncheon and Fashion Presentation, Produced and Sponsored by South Coast Plaza, Funds Transformative Duchenne Muscular Dystrophy Research NEWPORT BEACH, California (August 21, 2025) – CureDuchenne, a global nonprofit dedicated […]
Developing a novel therapeutic designed to protect muscle in Becker and Duchenne muscular dystrophy Watch HERE
Legacy Giving August is Make-a-Will Month — the perfect time to reflect on how you can protect what matters most: your loved ones and the causes that hold a special […]
In this webinar, Dr. Naz Dastgir, Executive Medical Director and Clinical Development Lead for RGX-202 at REGENXBIO, shared information on the active and enrolling AFFINITY DUCHENNE trial and how families […]
Solid Biosciences announced a widely anticipated update on their INSPIRE DUCHENNE Phase 1/2 clinical trial of SGT-003, their investigational micro-dystrophin gene therapy for Duchenne muscular dystrophy. What this means for […]
The FDA is continuing its review of Ataluren (Translarna), the first potential therapy specifically for individuals with nonsense mutation Duchenne (nmDMD). There is currently no set decision date. The application […]
When the Duchenne and Becker community speaks, the world listens — not just because of what you say, but because of how deeply it comes from the heart. In recent […]
Breaking News: Sarepta to resume shipments of ELEVIDYS for ambulatory individuals with Duchenne 07/28/25 5:09 PM EDT We are pleased to report that the US FDA has recommended that Sarepta […]
Precision BioSciences is developing a novel gene editing approach for individuals with dystrophin mutations in the “hot spot” region of exons 45-55, with the goal of submitting applications later this […]
