July 22, 2025

Update on ELEVIDYS Shipments in the U.S.

This evening, Sarepta Therapeutics issued a press release stating that it has voluntarily and temporarily paused all shipments of ELEVIDYS for Duchenne in the United States, effective close of business tomorrow, July 22, 2025. According…

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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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July 14, 2023

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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June 22, 2023

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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June 21, 2023

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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June 8, 2023

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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May 24, 2023

Sarepta Announces PDUFA Extension and Update for the FDA’s Review of SRP-9001

        Sarepta Therapeutics announced today that they received an update from the FDA  on the Biologics License Application (BLA) for SRP-9001. The PDUFA date reportedly has been extended to June 22, 2023. …

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May 19, 2023

SRP-9001: Submit Your Community Perspectives to FDA

On May 12, the FDA held an Advisory Committee meeting for SRP-9001, a gene therapy intended to treat Duchenne. The Advisory Committee meeting was used by FDA to obtain independent expert advice on scientific, technical,…

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May 12, 2023

A Community United: Thank You for Raising Your Voice During the SRP-9001 Advisory Committee

Today was a historic day for the Duchenne community: the first FDA Advisory Committee meeting for a gene therapy intended to treat Duchenne. This meeting was held as part of the regulatory review process for…

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May 10, 2023

May 12: Advisory Committee Meeting for SRP-9001 – Live Stream Information & Meeting Materials Released

The FDA has shared live stream access information for the May 12th Advisory Committee for SRP-9001 (delandistrogene moxeparvovec), Sarepta Therapeutics’s investigational gene therapy, which has applied for FDA approval through the Accelerated Approval pathway with…

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