October 21, 2022

WATCH: Edgewise Therapeutics – A Novel Approach to Protect Muscle in Duchenne & Becker (Webinar Recording)

PPMD was joined by Edgewise Therapeutics for a community webinar on October 19, 2022. Edgewise’s Chief Scientific Officer and Chief Medical Officer discussed the clinical development program for EDG-5506, a myosin modulator, in Becker and…

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October 18, 2022

Edgewise Announces 6 Sites Enrolling for CANYON, Phase 2 Trial of EDG-5506 in Becker

Edgewise has shared a community letter announcing that six sites are now open for enrollment for the Canyon trial for individuals living with Becker muscular dystrophy. Canyon is a Phase 2 trial of EDG-5506, a…

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October 17, 2022

The Duchenne Registry has three new surveys!

The Duchenne Registry, powered by PPMD, is the largest, most comprehensive patient-reported registry for Duchenne and Becker muscular dystrophy. With over 5,000 registrants, the Registry connects Duchenne and Becker patients with actively recruiting clinical trials…

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September 30, 2022

Solid Biosciences Acquires AavantiBio & Shares Update on Duchenne Gene Therapy Pipeline Programs

Today Solid Biosciences shared an update with the Duchenne community regarding their strategic development. Solid announced they have acquired AavantiBio, a gene therapy company with a focus on rare cardiomyopathies and Friedeich’s ataxia. While this…

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September 29, 2022

Sarepta Therapeutics Submits Biologics License Application (BLA) for SRP-9001

Sarepta Therapeutics today announced it has submitted a Biologics License Application (BLA) to the FDA for the accelerated approval of SRP-9001 to treat ambulant patients with Duchenne. SRP-9001 is an investigational gene transfer therapy intended to deliver…

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September 28, 2022

PepGen Reports Data from Phase 1 Trial of PGN-EDO51

PepGen today shared data from its Phase I trial of PGN-EDO51 in healthy volunteers. PGN-EDO51 is an PMO-exon skipping therapy bound to a peptide which improves uptake into muscle cells, and targets those amenable to…

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September 27, 2022

Avidity Biosciences Announces FDA Clearance of IND Application for AOC 1044

PPMD is excited to share that Avidity Biosciences has announced that they have received clearance from the FDA on their Investigational New Drug (IND) application for AOC 1044, an exon skipping therapy targeting those amenable to exon…

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September 27, 2022

Understanding Duchenne Gene Therapy Trials & Results Data

Understanding Gene Therapy Trials & Results Data At PPMD, one of our goals is to help families navigate the concerns and decisions that are part of Duchenne. Some of the most common questions we get…

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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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