April 28, 2025

The BELS Questionnaire: A New Tool to Support Your Child’s Emotional Wellbeing

PPMD is excited to share that a new tool, the BELS (Behavioral, Emotional, Learning, and Social) questionnaire, has been developed by Drs. Natalie Truba (Nationwide Children’s Hospital) and Molly Colvin (Massachusetts General Hospital) and was…

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March 18, 2025

A Safety Update on ELEVIDYS

We are deeply saddened by the loss of a young member of our community following treatment with ELEVIDYS. According to Sarepta, the individual with Duchenne received ELEVIDYS and later suffered from complications of acute liver…

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January 23, 2025

Update from PPMD’s 2025 Duchenne Healthcare Professionals Summit

PPMD recently convened over 360 healthcare professionals, researchers, and industry partners, including representatives from the FDA, NIH, CDC, and international representatives in one room to discuss the latest updates in dystrophinopathy care and research at…

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January 15, 2025

PPMD Hosts 2025 Duchenne Healthcare Professionals Summit in San Diego, California

PPMD is excited to host the sixth in-person Duchenne Healthcare Professionals Summit in San Diego, California, January 15-17. Since the Summit’s inception in 2018, the event has brought together thousands of healthcare providers, researchers, leaders…

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January 10, 2025

Introducing PPMD’s Dystrophinopathy Clinical Research Network (DCRN): Advancing the Research Evolution

In our rapidly evolving therapeutic landscape, there is a critical need for cohesive infrastructure that helps track what happens when individuals are on more than one therapy and how we can continue to evolve standards…

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December 17, 2024

PPMD Convenes GI, GU, and Swallowing Experts for Two-Day Workshop

Earlier this month, PPMD convened experts from across the country in Columbus, Ohio for a two-day workshop focused on the development of care standards for gastrointestinal, urological, and swallowing issues in Duchenne and Becker.  As…

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October 29, 2024

Flu Season Ready: Protecting Yourself and Your Family

With the start of school and the onset of winter, we can expect a rise in respiratory viruses. This year, it’s crucial to safeguard yourself, your children, and your family from all respiratory infections, particularly…

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September 27, 2024

PPMD Hosts Meeting Exploring the Case for Cardiac Transplantation in Duchenne

As part of our ongoing Cardiac Initiative, PPMD is proud to host the upcoming professional meeting, “Expanding Access to Heart Transplantation: Focus on Muscular Dystrophy,” taking place September 27-28, 2024, at Stanford University. This important…

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September 26, 2024

PPMD Joins Duchenne Parent Project aps Respiratory International Professional Care Meeting

Parent Project Muscular Dystrophy (PPMD) is pleased to announce our participation in the upcoming “Respiratory Assessment and Management of Duchenne Muscular Dystrophy: Current Standards and Future Directions” meeting, which will take place in Rome, Italy,…

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September 18, 2024

PPMD & Global Patient Advocacy Groups Award BIND Project $113,000 to Further Brain Involvement Research

PPMD is thrilled to announce that we have collaborated with a number of patient organizations around the globe to provide Leiden University Medical Center (LUMC), together with the Duchenne Data Foundation (DDF), funding to further…

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