December 21, 2023

PPMD & Community Partners Urge Timely, Transparent FDA Review of EMBARK Data

Today, PPMD joined the Best Day Ever Foundation, CureDuchenne, the Jett Foundation, the Little Hercules Foundation, The Muscular Dystrophy Family Foundation, and Team Joseph in sending a letter to the Federal Drug Administration (FDA) regarding…

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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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September 13, 2022

Sarepta Shares EMBARK Study (SRP-9001-301) Enrollment Update

Sarepta Therapeutics, in partnership with Roche, has announced the EMBARK study of delandistrogene moxeparvovec (SRP-9001) in participants with Duchenne is now fully enrolled. Delandistrogene moxeparvovec (SRP-9001) is an investigational gene transfer therapy intended to deliver…

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October 11, 2021

Sarepta Therapeutics holds SRP-9001 R&D Day

This morning, Sarepta Therapeutics held SRP-9001 Micro-dystrophin R&D Day, during which Sarepta shared information from all their trials involving SRP-9001.  New data from the 103 Study of SRP-9001 (ENDEAVOR) using their commercial material was presented….

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October 8, 2021

Sarepta SRP-9001 Micro-dystrophin Gene Therapy Update and R&D Day

On October 4th, Sarepta Therapeutics announced the initiation, in partnership with Roche, of a pivotal study of SRP-9001-301 (rAAVrh74.MHCK7.micro-dystrophin) also known as EMBARK.  Simultaneously, the company announced plans to host a community webinar with PPMD…

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October 4, 2021

Sarepta Therapeutics in partnership with Roche announced the initiation of the EMBARK clinical trial for their investigational gene therapy product SRP-9001

Sarepta Therapeutics, in partnership with Roche, has announced the initiation of study SRP-9001-301, also known as EMBARK, a pivotal study of SRP-9001 (rAAVrh74.MHCK7. micro-dystrophin) for the treatment of Duchenne muscular dystrophy. It is important to…

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