June 23, 2022

Sarepta Therapeutics Announces Clinical Hold on Study SRP-5051-201 (MOMENTUM)

This afternoon Sarepta Therapeutics, Inc. announced that the FDA has placed a clinical hold on SRP-5051 (vesleteplirsen) following a serious adverse event of hypomagnesemia (low magnesium). SRP-5051 is Sarepta’s next-generation peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO)…

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January 11, 2022

Sarepta Announces Topline Results from Part 2 of Study SRP-9001-102 Evaluating Duchenne Gene Therapy Candidate

Sarepta Therapeutics has shared an update from Part 2 of Study SRP-9001-102, an ongoing clinical trial evaluating Sarepta’s gene therapy candidate SRP-9001 in participants with Duchenne. SRP-9001 is an investigational gene transfer therapy intended to…

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January 5, 2022

Sarepta Therapeutics Shares MOMENTUM, Study SRP-5051-201, Update

Sarepta Therapeutics has shared an update that SRP-5051-201, the MOMENTUM study (NCT04004065), is now recruiting Part B. MOMENTUM is a Phase 2, two-part, multiple-ascending-dose study of SRP-5051 for dose determination, then dose expansion, in patients…

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October 18, 2021

WATCH: SRP-9001 Micro-Dystrophin Gene Therapy Clinical Update (Webinar Recording)

Last week, Parent Project Muscular Dystrophy was joined by Sarepta Therapeutics for a webinar to discuss the details of the EMBARK clinical trial (SRP-9001-301). If you missed the live event, the recording can be found below.*…

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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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September 27, 2021

Sarepta Therapeutics Plans to Initiate Part B of the MOMENTUM Study of SRP-5051 in Patients with Duchenne Following Positive Interactions with FDA

PPMD is pleased to share that Sarepta Therapeutics plans to initiate Part B of the MOMENTUM study of SRP-5051 in patients with Duchenne muscular dystrophy following positive interactions with the U.S. Food and Drug Administration…

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June 11, 2021

WATCH: SRP-9001 Micro-Dystrophin Gene Therapy Clinical Update (Webinar Recording)

WATCH: SRP-9001 Micro-Dystrophin Gene Therapy Clinical Update Parent Project Muscular Dystrophy was joined by Sarepta Therapeutics for a webinar update on June 2, 2021. Sarepta was invited to present recent updates from their ongoing clinical…

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May 3, 2021

Sarepta Therapeutics Reports Positive Clinical Results from Phase 2 MOMENTUM Study of SRP-5051

Sarepta has announced positive results from Part A of the MOMENTUM study (Study 5051-201), a Phase 2 clinical trial of SRP-5051, its next-generation treatment for individuals with Duchenne who are amenable to exon 51 skipping. Part…

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