September 14, 2022

WATCH: SRP-9001 Data Update from Studies 101, 102, and 103 (August 2022)

On August 24, 2022, PPMD was joined by Sarepta Therapeutics for a community webinar to present a SRP-9001 gene therapy data update from Studies 101, 102, and 103, following their recent presentation at a medical…

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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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September 12, 2022

Edgewise Announces 4-Month Interim Results from ARCH Open Label Study of EDG-5506 in Adults with Becker

Edgewise Therapeutics has released 4-month interim results from the ongoing ARCH open label study of EDG-5506 in adults with Becker muscular dystrophy. EDG-5506 is a small molecule, taken orally, that is intended to help protect muscle…

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September 7, 2022

Edgewise Announces FDA Authorization for Phase 2 Clinical Trial of EDG-5506 in Participants with Duchenne

Edgewise Therapeutics has announced the FDA has authorized the initiation of the LYNX Phase 2 clinical trial of EDG-5506 in participants with Duchenne. EDG-5506 is a small molecule, taken orally, that is intended to help protect muscle by reducing…

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September 7, 2022

The Duchenne Registry: Advancing Research for Female Carriers

In the past, there has not been a great deal of research focused on female carriers, but this is changing. The Duchenne Registry, started by PPMD in 2007, has always collected data on female carriers. In…

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September 7, 2022

World Duchenne Awareness Day 2022: Stories of #WomenAndDuchenne

World Duchenne Awareness Day 2022: #WomenAndDuchenne Each year, World Duchenne Awareness Day highlights a special theme for further education. This year that theme is Women & Duchenne to raise awareness about females living with ‘dystrophinopathy’ (the spectrum of muscle…

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September 6, 2022

Dyne Therapeutics Announces First Patient Dosed in Phase 1/2 DELIVER Clinical Trial of DYNE-251

Dyne has announced that the first participant has been dosed in the Phase 1/2 DELIVER clinical trial of DYNE-251 in participants with Duchenne amenable to skipping exon 51. Dyne-251 is an exon skipping product that combines…

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September 6, 2022

Dr. Mena Scavina: My Story as a Clinician #WomenAndDuchenne

World Duchenne Awareness Day 2022: #WomenAndDuchenne Each year, World Duchenne Awareness Day highlights a special theme for further education. This year that theme is Women & Duchenne to raise awareness about females living with ‘dystrophinopathy’ (the spectrum of muscle…

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September 6, 2022

Pat Furlong: My Story as a Mom, Finding the Power of ‘Us’ #WomenAndDuchenne

World Duchenne Awareness Day 2022: #WomenAndDuchenne Each year, World Duchenne Awareness Day highlights a special theme for further education. This year that theme is Women & Duchenne to raise awareness about females living with ‘dystrophinopathy’ (the spectrum of muscle…

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September 6, 2022

Mariana: My Story as a Carrier with Cardiac Concerns #WomenAndDuchenne

World Duchenne Awareness Day 2022: #WomenAndDuchenne Each year, World Duchenne Awareness Day highlights a special theme for further education. This year that theme is Women & Duchenne to raise awareness about females living with ‘dystrophinopathy’ (the spectrum of muscle…

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