October 7, 2022

38th Annual Friar Fight to End Duchenne Golf Tournament Raises Over $43,000 in the Fight to End Duchenne

The 38th Annual Friar Fight to End Duchenne Golf Tournament raised $43,712 for PPMD this year, bringing their total raised for our organization to $1,129,475! This event continually makes us proud, to see such wonderful…

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October 6, 2022

PPMD Submits Updated Community Draft Guidance for Duchenne, Becker, and Related Dystrophinopathies to FDA

An exciting update to the revised community-led Duchenne Guidance for FDA is that it has been officially submitted to the agency! As you know, this Community Draft Guidance will serve as an update to the…

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October 5, 2022

WATCH: Psychosocial Considerations for Women in Duchenne (Webinar Recording)

While we often think of the physical aspects of Duchenne and Becker muscular dystrophy, we don’t often talk about the emotional effect it has—especially for caregivers. As part of World Duchenne Awareness Day’s emphasis on…

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October 3, 2022

Race Athlete Spotlight: Stacey Korte

When friends join the Race to End Duchenne, we are all unstoppable. Meet Stacey Why did you choose to join the Race to End Duchenne team? I joined the Race to End Duchenne team because…

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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 29, 2022

Thanks to you PPMD continues forward in all areas of support

As Duchenne Action Month comes to an end, PPMD continues to celebrate the actions you take that make us strong. Thanks to the unwavering support of our community and incredible donors, we continue to forge…

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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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