July 2021

Dear U.S. Duchenne Community,

We have received inquiries about the enrollment status of the first three cohorts of Study 9001-103, an open label micro-dystrophin gene therapy study. At this time, the clinical trial sites involved in cohorts 1-3 are no longer accepting new patients for screening.

Families have been reaching out and expressing their interest in participating in a SRP-9001 gene therapy study. We recognize the unmet medical need that is driving this interest, and also recognize the urgency stated by so many families.

It is important to note that planned future trials for SRP-9001 will be larger in size and at a greater number of locations globally. We will be sharing details of future studies as soon as we can with the community. Information about our ongoing trials can be found on clinicaltrials.gov.

Thank you,

Sarepta Patient Affairs
Email: [email protected]