Defeat Duchenne Canada is pleased to be able to share with our Canadian Duchenne community the latest updates on the INSPIRE DUCHENNE and Phase 3 IMPACT DUCHENNE clinical studies, regulatory engagement, and the progress Solid Bio is making to advance SGT-003 for the Duchenne community

Solid Biosciences has dosed 53 boys with SGT-003, an investigational gene therapy for Duchenne muscular dystrophy, including a 6-month-old child who was the youngest to receive this type of therapy. The safety results so far have been encouraging, with no serious side effects observed.

The company is now testing the therapy in two clinical trials: INSPIRE DUCHENNE (Phase 1/2) and IMPACT DUCHENNE (Phase 3), which is currently enrolling patients in Australia and Canada with plans to expand to the United States, United Kingdom, and Europe. The trials were designed to gather information about how safe the therapy is and how well it works, with the goal of eventually getting approval from health authorities. Solid Biosciences continues to work with the FDA and remains committed to keeping the community informed every step of the way.

Full community letter and recent press release can be found below.  

Want to know more about Duchenne Research?

Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: