Gene therapy for Duchenne is centered on the goal of successfully introducing into a muscle cell the correct genetic code, or recipe, necessary to make the dystrophin protein. Because dystrophin is such a large protein, smaller versions (referred to as micro-dystrophins) are inserted into the delivery vehicle. Viral delivery using the adeno-associated virus (AAV) harnesses the virus’s natural ability to deposit genetic material right to the muscle cell nucleus. The result of this viral “infection” would be the successful recoding of each muscle cell in the patient’s body so that a smaller but functional dystrophin protein could be made.
A Phase 2 clinical trial is active, but no longer recruiting participants.
On March 19, 2020, Sarepta Therapeutics has provided an update addressing questions from the community regarding COVID-19, including updates on current clinical trials and accessing approved therapies. On April 13, 2020, Sarepta Therapeutics shared additional COVID-19 resources.
This program is sponsored by Sarepta Therapeutics and has been partially funded by Parent Project Muscular Dystrophy.
|A Randomized, Double-blind, Placebo-controlled Study of SRP-9001|
|Systemic Gene Delivery Clinical Trial for Duchenne Muscular Dystrophy|
Sarepta Presents at the PPMD 2019 Annual Conference
Jerry Mendell, MD (Nationwide Children’s) Presents at the PPMD 2018 Annual Conference
Webinar: Understanding Gene Therapy, Part 2 - Nationwide Children's HospitalAs part of our ongoing series on Understanding Gene Therapy, Dr. Jerry Mendell of Nationwide Children’s Hospital joined PPMD for a webinar on September 6, 2017 to discuss his upcoming gene therapy trial, including trial design, inclusion/exclusion criteria, and timelines. This webinar is the second in our series on Gene Therapy where we are bring leaders in gene therapy technology to you to discuss their latest projects and provide an update on this incredibly exciting and expanding field.