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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February 18, 2025

Solid Biosciences Shares Initial Data from INSPIRE DUCHENNE Study of Gene Therapy Candidate SGT-003

Solid Biosciences Inc. has announced positive initial data from the company’s Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003. SGT-003 is a gene therapy candidate for the treatment of Duchenne that delivers a microdystrophin via their…

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February 4, 2025

Cumberland Pharmaceuticals Announces Positive Results from Phase 2 FIGHT DMD Trial of Ifetroban for Treatment of Duchenne Cardiomyopathy

Cumberland Pharmaceuticals Inc. has announced positive top-line results from its Phase 2 FIGHT DMD trial evaluating ifetroban for the treatment of Duchenne cardiomyopathy. Ifetroban is an oral thromboxane receptor antagonist aimed at reducing inflammation and…

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January 27, 2025

Sarepta Therapeutics Announces Results from Part 2 of EMBARK Study of ELEVIDYS

Sarepta Therapeutics, Inc. has announced positive topline results from Part 2 of EMBARK, the company’s Phase 3 clinical study of ELEVIDYS. ELEVIDYS is a micro-dystrophin gene therapy used for the treatment of individuals aged four…

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January 23, 2025

Update from PPMD’s 2025 Duchenne Healthcare Professionals Summit

PPMD recently convened over 360 healthcare professionals, researchers, and industry partners, including representatives from the FDA, NIH, CDC, and international representatives in one room to discuss the latest updates in dystrophinopathy care and research at…

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January 17, 2025

Race to End Duchenne Kicks Off 20th Anniversary at Walt Disney World Marathon Weekend

The Race to End Duchenne program is celebrating our 20th anniversary in 2025, and we spent the weekend of January 8-12 kicking it off at the place where it all began – the Walt Disney…

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January 16, 2025

PPMD Advocacy in the 119th Congress and the 47th Administration

As the 119th Congress and the 47th Administration prepare to take office, PPMD is committed to furthering the needs of our community through comprehensive, creative, and tenacious advocacy on Capitol Hill, regardless of politics. Over…

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