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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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 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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