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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March 26, 2025

Wave Life Sciences Announces Positive Data from FORWARD-53 Clinical Trial

​Wave Life Sciences has announced encouraging interim results from its Phase 2 FORWARD-53 clinical trial of WVE-N531, an exon skipping oligonucleotide being investigated for the treatment of individuals with Duchenne who are amenable to exon…

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March 25, 2025

REGENXBIO Shares Positive Biomarker Data from AFFINITY DUCHENNE Trial of RGX-202 Gene Therapy

REGENXBIO Inc. has reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202, an investigational gene therapy product delivering a micro-dystrophin transgene via AAV8…

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March 18, 2025

A Safety Update on ELEVIDYS

We are deeply saddened by the loss of a young member of our community following treatment with ELEVIDYS. According to Sarepta, the individual with Duchenne received ELEVIDYS and later suffered from complications of acute liver…

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March 17, 2025

Key Updates from the 2025 MDA Clinical & Scientific Conference

The 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference is underway in Dallas, Texas, bringing together leading experts, researchers, and advocates in the neuromuscular disease community. Today, several significant announcements were made by companies…

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March 17, 2025

WATCH: Capricor Therapeutics – Update on Deramiocel for Cardiomyopathy in Patients with Duchenne (Webinar Recording)

Capricor Therapeutics recently joined PPMD for a community webinar to provide an update on their novel cell therapy, deramiocel (formerly CAP-1002), targeting cardiomyopathy in individuals living with Duchenne. The Capricor team discussed their ongoing Biologics…

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