November 25, 2025

PPMD Launches New Study to Understand Patient and Caregiver Treatment Preferences

PPMD has a long history of working alongside our community to document treatment preferences and share those insights with regulatory authorities such as the FDA. Through town halls, listening sessions, and conversations at community events,…

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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 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 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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October 24, 2025

Italfarmaco Addresses Patient Deaths, DUVYZAT Safety Profile in Community Letter

Italfarmaco, the company that developed DUVYZAT® (givinostat), has shared a letter to the Duchenne muscular dystrophy community addressing concerns raised in recent online, community-based discussions about patient deaths involving individuals living with Duchenne who were…

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October 22, 2025

Becker Muscular Dystrophy Series Part 1: Neuromuscular Care and Genetics

The first session in PPMD’s Becker muscular dystrophy webinar series brought together clinical experts and a community member to talk about genetics, neuromuscular care, and what it means to live with Becker. Below is an…

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October 17, 2025

Newly Published Study Evaluates Patients’ and Caregivers’ Acceptable Risk from Gene Therapy

A new threshold study, “Re-evaluating Acceptable Risk of Death from Gene Therapy,” has been published in the European Journal of Medical Genetics. This work addresses a critical question: how should risk and benefit be assessed…

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