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 4, 2025

The Future of Newborn Screening for Duchenne: Moving Forward After the ACHDNC’s Dissolution

Yesterday, we received news that the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) has been terminated, effective immediately. For over 20 years, this committee has served as the guiding body for adding…

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April 2, 2025

Recap: PPMD Together: Minneapolis, MN

This past weekend, PPMD was joined by families, individuals with Duchenne and Becker, clinicians, researchers, industry partners and local vendors for 2 days of community building, knowledge sharing, and meaningful connections at our PPMD Together…

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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 19, 2025

Duchenne Secures $12.5 Million in CDMRP Funding for FY25: A Hard-Fought Victory in a Challenging Year

We are humbled and grateful to share that Duchenne muscular dystrophy has been recommended by House and Senate Defense Appropriations leadership for  $12.5 million in federal funding for Fiscal Year 2025 (FY25) through the Congressionally…

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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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