We are pleased to share the Solid Biosciences community letter that provides data from their Phase 1/2 trial of SGT-003 showing increases in average microdystrophin expression of 110% and improvements in multiple muscle health biomarkers in the first people dosed.

SGT-003 is a gene therapy that uses an adeno-associated virus (AAV) to deliver a healthy, engineered (or man-made) version of the dystrophin gene to people with Duchenne muscular dystrophy (DMD). An AAV is a virus that does not cause illness; it just acts as delivery method to get the healthy gene into a person’s body. The goal of SGT-003 is to activate the dystrophin gene in people with DMD, with the hopes of being a cure to the disease.

Defeat Duchenne Canada looks forward to more updates on this important study. Please see Solid Biosciences full community letter and press release below.

Please join Defeat Duchenne Canada and members of the Solid Biosciences team to review initial positive data from the INSPIRE DUCHENNE study of SGT-003 on Tuesday, March 4, 2025 at 5:00 p.m. ET. Register at the link below.

Parent Project Muscular Dystrophy (PPMD) will host a community webinar with members of the Solid Biosciences team on Tuesday, February 25 at 1:00 PM ET. Register via the link below.

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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: