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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November 12, 2024

Calling All Adults with Duchenne & Becker: Join the 2025 PPMD Adult Advisory Committee

Have you been looking for a way to engage with the community more? Do you have a personal experience that you would like to share with the larger community or through federal and state advocacy…

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September 20, 2024

PPMD Contributes to National Academies Report on Rare Disease Regulatory Processes

We are proud to highlight the National Academies of Sciences, Engineering, and Medicine (NASEM) Consensus Study Report: Regulatory Processes for Rare Disease Drugs in the United States and European Union – Flexibilities and Collaborative Opportunities,…

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September 17, 2024

Navigating Access & Resources

Navigating Access & Resources Navigating insurance access for medical care such as approved therapies or equipment can be daunting. Parents, patients, and healthcare providers spend hours putting together documents and speaking on the phone trying…

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September 12, 2024

Watch the Recording: Advocating for A Successful School Year

Recently, PPMD’s Alexis Hazlett was joined by panelists Jodi Krause (Colorado Children’s Hospital), Jill Castle, and Patrick Moeschen to discuss advocating for your child throughout the school year. The speakers dove into the differences between…

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September 6, 2024

Raising Our Voices for Duchenne: Recap of PPMD’s Advocacy Webinar

On Thursday, September 5, 2024, PPMD hosted a special webinar, “PPMD Advocacy: Raising Our Voices in 2024 and Beyond,” bringing together advocates to reflect on PPMD’s advocacy efforts in our 30th anniversary year and discuss…

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September 4, 2024

Introducing PPMD’s Newborn Screening Action Center: A Hub for Advocacy and Education

We are thrilled to announce the launch of PPMD’s Newborn Screening Action Center, a powerful resource designed to both educate and empower you as we work to ensure that every baby, in every state, has…

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September 1, 2024

Raising Our Voices This Duchenne Action Month

PPMD is excited to 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…

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August 23, 2024

Duchenne Added to Massachusetts Newborn Screening Panel

PPMD is excited to announce another significant milestone: Massachusetts has officially approved the addition of Duchenne to the state’s newborn screening panel! The addition of Duchenne to the Commonwealth’s newborn screen panel was included as…

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August 12, 2024

PPMD Urges FDA to Conduct a Full Review of Ataluren (Translarna) Following Resubmission of NDA

PTC Therapeutics has shared that it has resubmitted its New Drug Application (NDA) to the FDA for review and potential approval of ataluren (Translarna). Ataluren is an oral small molecule treatment that allows for stop-codon…

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