August 4, 2025

Dyne Therapeutics, Inc. announced today FDA Breakthrough Therapy Designation for DYNE-251 in Duchenne Muscular Dystrophy

Dyne Therapeutics has announced FDA Breakthrough Therapy Designation for DYNE-251 in Duchenne Muscular Dystrophy. This designation reflects encouraging early results from the DELIVER clinical trial, where patients demonstrated sustained functional improvements, such as faster time…

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August 1, 2025

Watch: Capricor Therapeutics – Regulatory Update & Clinical Insights on Deramiocel for Duchenne Cardiomyopathy

Capricor Therapeutics recently joined PPMD for a community webinar on Tuesday, July 29, 2025 to discuss the current status of Capricor’s Biologics License Application (BLA) for Deramiocel (CAP-1002). We discussed the regulatory implications of a…

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July 23, 2025

FDA Grants Breakthrough Therapy Designation to Avidity’s del-zota

Avidity Biosciences, Inc. today shared that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to del-zota (AOC 1044) for the treatment of Duchenne in individuals amenable to exon 44 skipping.  Del-zota…

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July 11, 2025

Capricor Therapeutics Receives Complete Response Letter Regarding Deramiocel (CAP-1002)

On July 9th, the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for Capricor’s Biologics License Application (BLA) for Deramiocel (CAP-1002), an investigational cell therapy for Duchenne cardiomyopathy. This decision means…

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July 2, 2025

Webinar Recording: Edgewise Therapeutics – Update on Sevasemten Program in Becker and Duchenne

Edgewise Therapeutics recently joined PPMD for a community webinar for an update on the company’s sevasemten program in Duchenne and Becker. The Edgewise team reviewed new Open-label Extension MESA data in Becker, as well as…

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June 26, 2025

Edgewise Therapeutics Shares Positive Results on Sevasemten Program for Becker and Duchenne

Edgewise Therapeutics, Inc., has announced positive results in the company’s sevasemten program for both Becker and Duchenne. These data include results from: MESA, an open label extension trial of participants with Becker who were previously…

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June 15, 2025

A Safety Update on ELEVIDYS – June 2025

We are heartbroken to learn of the loss of another member of our community following treatment with ELEVIDYS. According to Sarepta, this represents the second reported case of acute liver failure in a non-ambulatory patient…

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June 10, 2025

Watch: Dyne Therapeutics — The Clinical Impact of the FORCE™ Platform for Duchenne (Webinar Recording)

Dyne Therapeutics recently joined PPMD for a community webinar on the DELIVER clinical trial evaluating DYNE-251. The Dyne team provided the latest updates on the Phase 1/2 global DELIVER study of the company’s investigational therapy…

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June 5, 2025

REGENXBIO Shares Positive Functional Data from Phase I/II AFFINITY DUCHENNE Trial of RGX-202

Today, REGENXBIO announced new data from its ongoing Phase I/II AFFINITY DUCHENNE® trial of RGX-202, an investigational gene therapy being developed for individuals with Duchenne muscular dystrophy. RGX-202 is designed to deliver microdystrophin via AAV8…

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May 29, 2025

PepGen Announces Discontinuation of PGN-EDO51 Development

We are disappointed to share that PepGen Inc. announced the company’s decision to end development of PGN-EDO51, its investigational therapy for the treatment of people with Duchenne amenable to exon 51 skipping. PepGen indicated that…

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