advocacy

PPMD Honored with RAPS 2026 Patient-Centered Health Award

PPMD is honored to receive the 2026 Patient-Centered Health Award from the Regulatory Affairs Professionals Society (RAPS), recognizing organizations that have made significant contributions to advancing patient-centered healthcare and ensuring patient perspectives inform the development…

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Preparing for the Deramiocel FDA Advisory Committee Meeting: How to Submit Written Testimony

The FDA has scheduled an Advisory Committee meeting for July 29, 2026 to review Deramiocel, Capricor’s investigational cell therapy for Duchenne muscular dystrophy. The meeting will be held in person with the option to participate…

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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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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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February 26, 2026

25 Years Later: Reflecting on the 2001 MD-CARE Act Hearing and Where We Are Now

On Thursday, February 26, advocates, clinicians, patient leaders, and policymakers came together for a powerful Senate Special Committee on Aging hearing titled “From Regulator to Roadblock: How FDA Bureaucracy Stifles Innovation.” This conversation served as…

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February 11, 2026

Rare Disease Advocacy Delivers: Major Federal Wins for Duchenne and Becker Community

We applaud a major legislative victory for the rare disease and Duchenne and Becker community — one made possible because advocates like you raised your voices, shared your stories, and sustained pressure on lawmakers. With…

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Budget Reconciliation Update and the Impact on Medicaid

PPMD is closely monitoring the U.S. House of Representatives Energy and Commerce Committee markup of the Committee’s budget reconciliation text, scheduled for tomorrow, Tuesday, May 13, 2025, at 2:00 PM ET. Updated legislative language released…

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Watch: Duchenne Newborn Screening – Next Steps Following ACHDNC Dissolution (Webinar Recording)

Last week, PPMD and the Muscular Dystrophy Association provided insights into the current status of the Duchenne nomination to the federal Recommended Uniform Screening Panel (RUSP) for newborn screening following the dissolution of the Advisory…

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