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FDA Announces Advisory Committee Meeting to Review Capricor Therapeutics’ Biologics License Application for Deramiocel

The U.S. Food and Drug Administration (FDA) has scheduled an Advisory Committee meeting for July 29, 2026, to review Capricor Therapeutics’ Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for the treatment of…

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Inside the HHS Clinical Trials Roundtable: Why Speed, Access, and Rigor Matter for the Duchenne Community

On June 22, 2026, PPMD Chief Executive Officer Katherine Beaverson, MS attended the U.S. Department of Health and Human Services (HHS) Clinical Trials Roundtable in Washington, D.C., joining leaders from across government, industry, academia, and…

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Massachusetts Begins Newborn Screening for Duchenne

As of today, Massachusetts has officially begun screening all newborns for Duchenne muscular dystrophy. This milestone comes after the passage of the state’s Maternal Health bill (H. 4999), which was signed into law by Governor…

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PPMD Joins FDA Leadership for Rare Disease Roundtable

On Wednesday, June 3rd, PPMD was invited to participate in a closed roundtable with senior leadership at the U.S. Food and Drug Administration (FDA). The meeting was convened by Amy Comstock Rick, JD, Director of…

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Momentum Continues: Duchenne Newborn Screening Advances Across the US

Duchenne was added to the Recommended Uniform Screening Panel (RUSP) (RUSP) on December 16, 2025, and in the months since, that decision, coupled with our continued advocacy efforts, have driven meaningful action at the state…

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Advancing Duchenne Newborn Screening: A Collaborative Path Forward

On April 24, 2026, PPMD, in collaboration with the Muscular Dystrophy Association (MDA) and the Little Hercules Foundation (LHF), convened a Duchenne Newborn Screening (NBS) Stakeholder Meeting in Philadelphia. The meeting’s main goal was to…

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PTC Therapeutics Shares Updated Community Letter Regarding Ataluren

Today PTC Therapeutics released an updated letter to the Duchenne community on their website with additional details following the company’s withdrawal of the ataluren New Drug Application (NDA). This update includes new information on a…

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Community Comes Together on Capitol Hill: A Recap of PPMD’s 2026 Advocacy Conference

Each year, the Duchenne and Becker muscular dystrophy community comes together in Washington, DC with a shared mission: to ensure that the voices of individuals living with Duchenne and Becker and their families are heard…

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PPMD and FED Announce 2026 Advocacy Leadership Awards Honoring Bipartisan Champions in Congress

PPMD, in partnership with the Foundation to Eradicate Duchenne (FED), is pleased to announce Representative Troy Balderson (OH-12), Senator Susan Collins (ME), Senator Amy Klobuchar (MN), Representative Doris Matsui (CA-07), and Senator Roger Wicker (MS)…

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Advocates Unite in Washington to Advance Duchenne and Becker Policy, Mark 25th Anniversary of MD-CARE Act

PPMD heads to Capitol Hill today with a group of more than 120 Duchenne and Becker advocates to meet with Congressional leaders during this year’s annual PPMD Advocacy Conference. “Together, our community has secured victories…

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