Blog

PPMD’s Certified Care Center Program

PPMD continues to advocate to ensure families navigating a diagnosis of Duchenne and Becker muscular dystrophy have access to optimal care for their loved ones. The Certified Duchenne Care Center Program (CDCC Program), a unique…

Learn More

Steroids and Stress Dosing: Understanding the Updated PJ Nicholoff Steroid Protocol

Last week, PPMD brought together leading experts to address steroids, stress dosing and the updated PJ Nicholoff Steroid Protocol. Drs. Aravindhan Veerapandiyan, David Weber, and Anne Marie Sbrocchi shared valuable insights on the management of…

Learn More

Florida Signs Duchenne Newborn Screening into Law

We are delighted to share exciting news for the Duchenne community: Governor Ron DeSantis has officially signed Florida’s Duchenne newborn screening legislation (HB 1089 / SB 524) into law. With this historic act, Florida becomes…

Learn More

A Decade of Impact: Honoring PJ’s Protocol and Its Legacy

Ten years ago, the Duchenne community gained a vital tool for the clinical management of adrenal insufficiency and steroid stress dosing for individuals on long-term corticosteroid therapies: the PJ Nicholoff Steroid Protocol for Healthcare Providers….

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

Join Our Mailing List