November 20, 2025

Closing Out the Year with Connection & Community at PPMD Together

Recently, the Duchenne and Becker community gathered virtually for the final PPMD Together meeting of the year. This intimate session centered on fostering friendships for everyone in the family while living with dystrophinopathy.  The meeting,…

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November 19, 2025

Avidity Biosciences Announces U.S. Managed Access Program for del-zota

Avidity Biosciences, Inc. has announced its Managed Access Program (MAP) for delpacibart zotadirsen (del-zota) for eligible individuals living with Duchenne who are amenable to exon 44 skipping in the United States. MAPs allow a limited…

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November 18, 2025

Becker Muscular Dystrophy Series – Part 2: Cardiopulmonary Care

The second session in PPMD’s Becker Muscular Dystrophy webinar series focused on cardiopulmonary care. Our clinical experts reviewed what every person living with Becker and their families should know about care for the heart and…

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November 14, 2025

FDA Approves New Safety Warning and Revised Indication for ELEVIDYS

Today the FDA announced a significant update to the labeling and indication of ELEVIDYS for the treatment of individuals living with Duchenne following reports of death based on acute liver failure (ALF) in non-ambulatory patients…

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November 14, 2025

PPMD Continues to Drive Progress with Your Support

PPMD is committed to every future, working toward the day when every single family has options for, and access to, therapies and specialized care. In 2025, thanks to your support, families are receiving earlier diagnoses,…

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November 10, 2025

Tips for Smooth Holiday Travel

The holidays are a special time to connect with family and friends, and for many, that involves travel. To help make your journey a little easier, PPMD has gathered some helpful travel tips and resources!…

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November 7, 2025

Welcoming the New Members of PAAC for Fall 2025

PPMD’s Adult Advisory Committee (PAAC) recently expanded with 19 inspiring new members, bringing the committee to its largest size ever—73 members strong. These incredible individuals bring diverse academic backgrounds ranging from marketing and communication to…

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November 6, 2025

Advancing Brain and Behavior Care in Dystrophinopathy

Recently, PPMD led a meeting with experts from across the US and Europe to discuss the evolving understanding of brain and behavior implications in individuals with dystrophinopathy. The two day workshop, led by Drs. Molly…

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November 5, 2025

Understanding Fat Embolism Syndrome (FES) and Protecting Bone Health

PPMD recently hosted a webinar about Fat Embolism Syndrome (FES), covering what FES is, what symptoms to watch for, and how to help keep people with dystrophinopathy safe and healthy. Four clinical experts shared practical…

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November 5, 2025

Solid Biosciences Shares Interim Data from INSPIRE DUCHENNE Trial of SGT-003

Solid Biosciences Inc. has announced positive new interim data from the Phase 1/2 INSPIRE DUCHENNE clinical trial and provided an update on its planned meeting with the U.S. Food and Drug Administration (FDA) to discuss…

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