- Sarepta and its partner Roche present new results and analyses at the International Congress on Neuromuscular Diseases (ICNMD), which demonstrate that SRP-9001 shows consistent, statistically significant functional benefits in individuals with Duchenne versus a propensity-weighted external control that continue to positively diverge from natural history disease course
- In the 20-patient Cohort 1 of SRP-9001-103 (ENDEAVOR), SRP-9001-treated participants improved 4 points from their pre-therapy baselines, and 3.8 points (unadjusted means) and 3.2 points (least squared means) at 52 weeks on the North Star Ambulatory Assessment (NSAA) compared to a propensity-weighted external control group (p=
Sarepta Therapeutics’ Investigational Gene Therapy SRP-9001 for Duchenne Muscular Dystrophy Demonstrates Significant Functional Improvements Across Multiple Studies
On 6ix: 6ix.com/news/sarepta-therapeutics-investigational-gene-therapy-srp-9001-for-duchenne-muscular
Watch Sarepta Therapeutics' latest callSarepta Therapeutics (NASDAQ:SRPT) Q2 2026 Earnings Call · Replay · Aug 5Read the transcript of Sarepta Therapeutics (NASDAQ:SRPT) Q2 2026 Earnings CallSarepta Therapeutics, Inc. on 6ix
Story so far
- Sarepta Therapeutics’ Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy, SRP-9001, Demonstrates Robust Expression and Consistent Safety Profile Using Sarepta’s Commercial Process Material
- Sarepta Therapeutics’ Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Expression and Functional Improvements 2 Years After Administration
- Sarepta Therapeutics Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Functional Improvements 18-months After Administration
Earlier from Sarepta Therapeutics
- Sarepta Therapeutics to Share New Clinical Data and Integrated Analysis for SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy on July 6, 2022 at 8: 30am ET
- Sarepta Therapeutics Provides Update on SRP-5051 for the Treatment of Duchenne Muscular Dystrophy
- Sarepta Therapeutics to Present at the Goldman Sachs 43rd Annual Global Healthcare Conference
- Sarepta Therapeutics Appoints Michael Chambers and Kathryn Boor, Ph.D., to Its Board of Directors
- Sarepta Therapeutics to Present at the BofA Securities 2022 Healthcare Conference
Later from Sarepta Therapeutics
- Sarepta Therapeutics to Announce Second Quarter 2022 Financial Results and Recent Corporate Developments on August 2, 2022
- Sarepta Therapeutics Announces Intent to Submit an Accelerated Approval Biologics License Application for its Gene Therapy SRP-9001 to Treat Duchenne Muscular Dystrophy
- Sarepta Therapeutics Announces Second Quarter 2022 Financial Results and Recent Corporate Developments
- Sarepta Therapeutics Announces Progress on the MyoAAV Program and Exclusive Licensing Agreement with The Broad Institute for MyoAAV Next-generation Capsids for Rare Genetic Diseases
- Sarepta Therapeutics Announces That FDA has Lifted its Clinical Hold on SRP-5051 for the Treatment of Duchenne Muscular Dystrophy
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