September 21, 2022

WATCH: Medical Management of Women & Carriers (Webinar Recording)

WATCH: Medical Management of Women & Carriers (Webinar Recording) As part of World Duchenne Awareness Day’s emphasis on Women in Duchenne, PPMD recently hosted a webinar titled, The Medical Management of Women Carriers of Duchenne….

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September 16, 2022

ImagingNMD: Expanding Natural History Studies in Duchenne, Becker, and Carriers

Are you interested in contributing to clinical research to understand how muscle changes over time in Duchenne, Becker, and manifesting carriers? University of Florida’s ImagingNMD group has several studies currently recruiting participants to better understand…

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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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