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EMBARK, gene therapy for the treatment of DMD

ENDEAVOR

Sarepta Therapeutics announces initiation of EMBARK, a global p0ivotal study of SRP-9001, gene therapy for the treatment of DMD (Duchenne Muscular Dystrophy) Original press release > CAMBRIDGE, Mass., Oct. 04, […]

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Part B of MOMENTUM study of SRP-5051 in patients with DMD

MOMENTUM study of SRP-5051

Sarepta Therapeutics to initiate Part B of MOMENTUM study of SRP-5051 in patients with Duchenne muscular dystrophy (DMD) amenable to Exon 51 skipping following positive interactions with FDA Original press […]

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World Duchenne Awareness Day 2021

September 7 is World Duchenne Awareness Day. On this day, we raise awareness for Duchenne and Becker muscular dystrophy (DMD and BMD) around the globe. An initiative coordinated by the […]

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ENDEAVOR, gene therapy for DMD

ENDEAVOR

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.   The first 11 participants […]

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Positive clinical results from phase 2 MOMENTUM study

Sarepta Therapeutics reports positive clinical results from phase 2 MOMENTUM study of SRP-5051 in patients with Duchenne muscular dystrophy amenable to skipping exon 51 Results suggest a highly potent next-generation […]

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Genethon announces First Patient dosed in Clinical Trial

genethon gene therapy Généthon annonce le traitement d’un premier patient

Genethon announces First Patient dosed in Clinical Trial of Investigational Gene therapy GNT004 for Duchenne Muscular Dystrophy. Communiqué de presse original > Genethon   A first participant was dosed at I-Motion, […]