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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May 22, 2025

Satellos Announces Positive Functional Data from Phase 1b Open-Label Trial of SAT-3247 in Adults with Duchenne

Satellos Bioscience Inc. has announced encouraging data from the company’s Phase 1b open-label study of SAT-3247, an oral small molecule inhibitor of AAK, designed to address progressive muscle loss by restoring muscle regeneration in response…

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May 1, 2025

Share your NOW: The impact of up-to-date data in The Duchenne Registry

The Duchenne Registry is designed as a longitudinal (long-term) research study, collecting information from the same people about the same topics over time. Data entered at one point in time is just that – a…

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April 28, 2025

Watch: Sarepta Therapeutics – Duchenne Gene Therapy Update (Webinar Recording)

Sarepta Therapeutics recently joined PPMD for a community webinar to share the latest updates on their gene therapy program. In addition to program updates, Dr. Chet Villa, cardiologist at Cincinnati Children’s Hospital, and Dr. Craig…

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April 8, 2025

Watch: Wave Life Sciences – Update on FORWARD-53 Study of WVE-N531 (Webinar Recording)

Wave Life Sciences recently joined PPMD for a community webinar to provide an update on the company’s Phase 2 FORWARD-53 trial of WVE-N531, Wave’s exon skipping oligonucleotide being investigated for the treatment of individuals with…

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April 1, 2025

Watch: Avidity Biosciences – Topline Data from EXPLORE44® Clinical Trial (Webinar Recording)

Avidity Biosciences recently joined PPMD for a community webinar to share topline data from their EXPLORE44® clinical trial, which is assessing the safety and efficacy of the investigational therapy delpacibart zotadirsen (formerly AOC 1044, abbreviated…

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March 29, 2025

Dr. Peter Marks Resigns from FDA Center for Biologics Evaluation and Research

PPMD is deeply disappointed that Dr. Peter Marks has made the decision to leave his role as Director of the FDA’s Center for Biologics Evaluation and Research (CBER). In his resignation letter, addressed to Acting…

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