Solid Biosciences has sent a community letter announcing that not only has dosing of SGT-003 begun at their clinical trial sites in the United States, but that the trial will expand to a site in Canada soon.
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 personโs 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
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:
