Early Phase Study of SRP-9001 in Boys with Duchenne

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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 full details on eligibility criteria and contact information, please see more information on the Clinical Trials website: https://clinicaltrials.gov/ct2/show/NCT04626674

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.

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