Solid Biosciences, the developer of the gene therapy SGT-003, has highlighted results of its INSPIRE DUCHENNE clinical trial, which is ongoing worldwide, including an active clinical site at The Hospital for Sick Children in Toronto, Ontario.

As of August 12, 2025^, the therapy was administered to 15 patients living with Duchenne muscular dystrophy in the United States and appears to be well-tolerated. One patient experienced mild liver symptoms but is now asymptomatic without change to daily activity. 

Building off their early trial results, Solid plans to treat more than 20 additional patients by year’s end. The company also plans to meet with the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2025 to discuss regulatory approval in the USA. Lastly, around the same time, Solid plans to initiate a separate, randomized, placebo-controlled trial evaluating SGT-003 outside the U.S. to build a case for approval in other countries, supporting potential global regulatory authorizations as they strive to bring their gene therapy to those who need it globally.

Defeat Duchenne Canada looks forward to more updates on this important study. Please see Solid Biosciences’ full community letter and press release below. You can also watch our latest update about the INSPIRE DUCHENNE study from March 4, 2025, with Solid Biosciences on our YouTube channel.

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: