Blog

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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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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December 16, 2025

Historic Victory: Duchenne Added to the Recommended Uniform Screening Panel (RUSP)

Today marks a monumental victory for the Duchenne community. The U.S. Department of Health and Human Services (HHS) has officially added Duchenne to the Recommended Uniform Screening Panel (RUSP)—the national list of conditions recommended for…

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December 5, 2025

Honoring Every Journey in Duchenne and Becker: Advancing Therapies, Access, and Care For All

For more than three decades, Parent Project Muscular Dystrophy (PPMD) has been committed to bringing therapies closer to, and improving care for, every person living with Duchenne and Becker muscular dystrophy. The Duchenne and Becker…

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October 10, 2025

Standing Together for Access: Ensuring Duchenne Patients Can Benefit from Approved Therapies

PPMD has always fought to ensure that every individual living with Duchenne has access to safe, effective, FDA-approved treatments. This week, PPMD joined six partner organizations: Best Day Ever Foundation, Charlie’s Cure, CureDuchenne, Little Hercules…

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September 5, 2025

PPMD Kicks Off World Duchenne Awareness Day with Bad Shirt Friday, Special United Nations Documentary Premiere

PPMD will join forces with fellow Duchenne-specific organizations from around the world to celebrate World Duchenne Awareness Day on September 7. This global initiative, spearheaded by the World Duchenne Organization, aims to raise awareness about Duchenne and…

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