Today we announced early clinical results from Phase 1/2 ascending-dose studies of SRP-1001 for FSHD1 and SRP-1003 for DM1. The results demonstrated dose-dependent muscle exposure, early biomarker effects and favorable tolerability, reinforcing confidence in the potential for differentiated delivery capabilities of our platform. Learn more: https://t.co/PGXyrSg0ju
On Wednesday, March 25 at 8:30 am Eastern, we will host a live webcast to discuss first clinical results from two of our siRNA programs for neuromuscular diseases. Tune in here: https://t.co/f4v5lc6rqd
News: Today we shared an update on our ongoing interactions with the U.S. FDA and intention to submit sNDAs seeking conversion of accelerated approvals to traditional approvals by the end of April. Visit our website for more information.
Happy to be here in Orlando at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference sharing updates and connecting with the community. Come find us! #MDAconference#MDA
@OmarAbb56696664 Hello, Omar. If you have questions, it's best to speak directly with your healthcare provider. You also can email our Patient Affairs team at [email protected]. Thank you!
Excited to be a partner of the 2026 Muscular Dystrophy Association Clinical & Scientific Conference. We’ll be there sharing updates on how Sarepta is advancing science and generating new insights for patients and healthcare providers. #MDAconference
@DhandaBihari Hello, and thank you for your message. Please contact your healthcare provider to discuss medical questions. If you have additional questions, you can email our Patient Affairs team at [email protected]. Thank you.
Today we announced positive topline three-year EMBARK results showing that our approved gene therapy significantly slows disease progression on key functional measures in ambulatory Duchenne patients. Read the release at https://t.co/Fj9GrqIHK8.
Today at #JPM26, CEO Doug Ingram discussed our preliminary Q4 and full-year 2025 performance and shared an update on corporate developments. Read more: https://t.co/NLgt8z91sK
Sarepta announced the submission of its CTA for Study SRP-1005-101 to the New Zealand Medicines and Medical Devices Safety Authority. SRP-1005 is an investigational small interfering RNA (siRNA) therapeutic for the treatment of Huntington’s Disease. Read more: https://t.co/KagZn0KIGY
Big news: Duchenne muscular dystrophy is now on the Recommended Uniform Screening Panel (RUSP)! This milestone reflects tireless advocacy from families, organizations and leaders committed to early diagnosis. Learn more: https://t.co/n0bLxnS884
#Newbornscreening
We just announced that the U.S. FDA approved to begin dosing in Cohort 8 of the ENDEAVOR study to evaluate the use of an enhanced immunosuppressive regimen as part of treatment with our gene therapy for non-ambulatory patients. Read the press release: https://t.co/8h4x7YX4dZ
@WaleedWali19 Hello Waleed, and thank you for your message. Please contact your healthcare provider to discuss medical questions. If you have additional questions, you can email our Patient Affairs team at [email protected]. Thank you!
Today we shared an update on the prescribing information for our approved gene therapy. Read the press release: https://t.co/jclQqXfk54
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