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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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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March 14, 2025

The Impact of the Continuing Resolution and Federal Budget Cuts to the CDMRP on Duchenne Research

Today, the Senate passed a Continuing Resolution (CR) that includes a 57% cut to the Congressionally Directed Medical Research Program (CDMRP). While the CR may keep the government running in the short term, it brings…

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March 10, 2025

Advocates Unite in Washington D.C. to Champion Duchenne Policy Reforms

PPMD, with a group of more than 150 advocates, heads to Capitol Hill today to meet with Congressional leaders during this year’s annual PPMD Advocacy Conference. This marks the 23rd year that PPMD advocates will…

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March 4, 2025

Capricor Therapeutics Announces FDA Acceptance and Priority Review of Biologics License Application for Deramiocel

PPMD is excited to learn that the FDA has accepted Capricor Therapeutics’ Biologics License Application (BLA) for review, seeking traditional approval for deramiocel (CAP-1002). Additionally, the FDA granted the BLA Priority Review, reducing the review…

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February 28, 2025

WATCH: Solid Biosciences – Update on Gene Therapy Candidate SGT-003 (Webinar Recording)

Members of the Solid Biosciences team recently joined PPMD for a community webinar to review initial positive data from the INSPIRE DUCHENNE study of SGT-003, a next-generation microdystrophin gene therapy candidate. The Solid team provided…

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