A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From SRP-9001 in Participants With Duchenne Muscular Dystrophy
Sarepta is now expanding their trial of SRP-9001 (ENDEAVOR) to include 3-year-old boys living with Duchenne muscular dystrophy for participation in an early phase clinical trial to evaluate the efficacy of Sarepta’s investigational gene replacement therapy.
SRP-9001 is administered as an intravenous infusion and uses an adeno-associated virus to deliver mini-dystrophin, a shortened version of the dystrophin gene, into the muscle tissue of boys with Duchenne. Treatment with SRP-9001 has the potential to lead to functional improvements in boys living with Duchenne.
At this time, studies will take place in the following United States locations:
- Stanford University
Palo Alto, California, United States, 94304
- University of California, Davis
Sacramento, California, United States, 95616
- Washington University in St. Louis
Saint Louis, Missouri, United States, 21205
- Nationwide Children’s Hospital
Columbus, Ohio, United States, 43205
- Children’s Hospital of The King’s Daughters
Norfolk, Virginia, United States, 23507
For more information about Sarepta’s investigative clinical trials, please contact: Advocacy@Sarepta.com
If you have questions, need help navigating clinical trials or want to schedule a 1:1, email us at Cares@CureDuchenne.org to set up a time that works with your schedule.