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Sarepta Therapeutics, Inc.

Pipeline8 assets

  • SRP-1001SRP-1001 / FSHD1 / siRNA / Neuromuscular
    Facioscapulohumeral muscular dystrophy, Type 1 (FSHD1)
  • SRP-1003SRP-1003 / DM1 / siRNA / Neuromuscular
    Myotonic dystrophy, Type 1 (DM1)
  • SRP-1002SRP-1002 / siRNA / Pulmonary
    Idiopathic pulmonary fibrosis (IPF)
  • SRP-1004SRP-1004 / siRNA / Neurology
    Spinocerebellar ataxia type 2 (SCA2)
  • SRP-1005SRP-1005 / siRNA / Neurology
    Huntington's disease (HD)
  • SRP-1007SRP-1007 / siRNA / Neurology
    Spinocerebellar ataxia type 1 (SCA1)
  • SRP-1006SRP-1006 / siRNA / Neurology
    Spinocerebellar ataxia type 3 (SCA3)
  • bidridistrogene xeboparvovecSRP-9003 / LGMD2E/R4 β-sarcoglycan / Gene Therapy / Neuromuscular
    Limb-girdle Muscular Dystrophy

Latest news12 items

All news

Sarepta Therapeutics to Announce First Quarter 2026 Financial Results

CAMBRIDGE, Mass. --(BUSINESS WIRE)--Apr. 22, 2026-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will report first quarter 2026 financial results after the Nasdaq Global Market closes on Wednesday, May 6, 2026. Subsequently, at 4:30 p.m.

Sarepta to Share First Clinical Data from siRNA Pipeline Targeting FSHD1 and DM1

CAMBRIDGE, Mass. --(BUSINESS WIRE)--Mar. 24, 2026-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Wed., March 25, 2026, at 8:30 am Eastern Time, the Company will host a webcast and conference call to present the early

Results Call • Phase 1/2

Sarepta Provides Regulatory Update on AMONDYS 45® and VYONDYS 53®

– Following feedback from FDA, Company intends to submit supplemental new drug applications to FDA by the end of April 2026 requesting conversion to traditional approval CAMBRIDGE, Mass. --(BUSINESS WIRE)--Mar. 19, 2026-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic

Sarepta Announces that Screening and Enrollment are Underway in ENDEAVOR Cohort 8 to Evaluate Enhanced Immunosuppression Regimen as Part of ELEVIDYS Gene Therapy for Non-Ambulant Individuals with Duchenne

- Approximately 25 non-ambulatory participants will receive sirolimus as part of the regimen in Cohort 8 of the ENDEAVOR study - The enhanced immunosuppressive regimen is designed to mitigate the risk of acute liver injury (ALI) and acute liver failure (ALF) associated with AAV gene therapy in

Trial Start • Phase 1

Sarepta Therapeutics to Present New Long-Term and Safety Data Across Gene Therapy and Exon-Skipping Programs at 2026 Muscular Dystrophy Association Clinical & Scientific Congress

Several abstracts, including a late-breaking podium presentation and posters, bring forward accumulating long-term efficacy, safety and caregiver-reported insights that deepen understanding of dystrophin restoration and its impact in Duchenne CAMBRIDGE, Mass. --(BUSINESS WIRE)--Feb.

Presentation / Abstract • Phase 3

Sarepta Therapeutics Announces Commercial Launch of ELEVIDYS in Japan

- Japan now offers ELEVIDYS gene therapy to children with Duchenne muscular dystrophy aged 3 years to less than 8 years - Company is eligible to receive a $40 million milestone payment upon first commercial sale in Japan CAMBRIDGE, Mass. --(BUSINESS WIRE)--Feb. 24, 2026-- Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc. • Endpoint Arena