Defeat Duchenne Canada is pleased to report that Solid Biosciences plans to expand its ongoing trial to include SickKids as a clinical trial site. Full Community Letter and recent Press Release can be found below.

INSPIRE DUCHENNE is a first-in-human, multicenter, Phase 1/2 open-label study investigating the safety, tolerability, and efficacy of a single infusion of SGT-003 in ambulant boys with Duchenne muscular dystrophy (DMD). SGT-003 is a gene therapy that uses an adeno-associated virus (AAV; a virus that does not cause illness) to deliver a healthy, engineered (or man-made) version of the dystrophin gene to people with DMD. 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.

“We are highly encouraged by the progress we have made with the INSPIRE DUCHENNE clinical trial over the past quarter … SGT-003 continues to be well tolerated in the first three patients dosed. As a result of encouraging early results observed in these patients, we have implemented an updated study protocol amending the clinical trial across key parameters, including enrollment size, age range, and clinical endpoint timelines.”

Bo Cumbo, President and CEO, Solid Biosciences.

The number of people with DMD included will now be 43 enrolled in the study and the age range broadened from the previous 4 to <8 years to 4 to <12 years.

Defeat Duchenne Canada looks forward to our upcoming webinar with representatives from Solid Bioscience on Wednesday, November 13 at 1pm Eastern. Please join us by registering through the link below.

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