sarepta therapeutics

FDA Expands ELEVIDYS Label Indication for Ages 4 Years and Older

PPMD is excited to share that the FDA has made the decision to expand the labeled indication for ELEVIDYS, a micro-dystrophin gene therapy used to treat people with Duchenne muscular dystrophy, to include individuals at…

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WATCH: Sarepta Therapeutics – Data update from MOMENTUM study, SRP-5051 (Webinar Recording)

Sarepta Therapeutics recently joined PPMD for a community webinar: Data Update From the MOMENTUM Study of SRP-5051. Sarepta Therapeutics was invited to share data from Part B of MOMENTUM, a Phase 2 Study of SRP-5051…

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January 29, 2024

Sarepta Therapeutics Reports Positive Data from Part B of MOMENTUM Study of SRP-5051

Sarepta Therapeutics, Inc. today announced positive data from Part B of SRP-5051-201, the MOMENTUM study. MOMENTUM is a global, Phase 2, multi-ascending dose clinical trial of SRP-5051 that enrolled patients aged 8 to 21 years….

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December 22, 2023

Sarepta Therapeutics Submits Efficacy Supplement to Expand the ELEVIDYS Label

Sarepta Therapeutics, Inc. today announced the submission of an efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS to expand its labeled indication. The company also shared that it completed the Phase 3 EMBARK…

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November 13, 2023

WATCH: Overview of Topline Results from Phase 3 EMBARK Study (Webinar recording)

PPMD recently held a community webinar to discuss the recently shared Phase 3 topline results of the EMBARK study (SRP-9001-301). Crystal Proud, MD, provided an overview of the study topline results, highlighting that EMBARK achieved…

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October 30, 2023

Sarepta Therapeutics Announces Topline Results from Phase 3 EMBARK Study of ELEVIDYS Gene Therapy

Sarepta Therapeutics has announced Phase 3 topline results of their EMBARK study with ELEVIDYS, a micro-dystrophin gene therapy product currently approved for patients aged 4-5 living with Duchenne. Sarepta shares that while EMBARK did not…

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WATCH: ELEVIDYS: What This Means for You and Your Family (Webinar Recording)

In case you missed it, PPMD was joined by Drs. Russ Butterfield of University of Utah and Sue Apkon of Colorado Children’s earlier this week to talk through some of the common questions around the…

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FDA grants approval via accelerated approval pathway to ELEVIDYS: First-ever gene therapy for individuals with Duchenne ages 4-5

Today is an incredible milestone for the entire Duchenne community. It is a first step. But a huge step forward. Today, the FDA has granted accelerated approval to ELEVIDYS (delandistrogene moxeparvovec-rokl), a micro-dystrophin gene therapy…

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Sarepta webinar recording

WATCH: ENVISION Study Update With Sarepta Therapeutics (Webinar Recording)

Last week Sarepta Therapeutics joined PPMD to discuss ENVISION, their upcoming 18-month placebo-controlled Phase 3 study of SRP-9001 (delandistrogene moxeparvovec) for older ambulatory and non-ambulatory individuals with Duchenne. Matthew Furgerson, PhD, Medical Director, Clinical Development…

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Sarepta Announces Update on ENVISION Study Enrollment in U.S.

Today, Sarepta Therapeutics announced information regarding their ENVISION study for SRP-9001. ENVISION is a 72-week placebo control design with crossover study of SRP-9001 (delandistrogene moxeparvovec) in older ambulatory and non-ambulatory individuals living with Duchenne muscular…

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