Blog

Sarepta to Resume ELEVIDYS Shipments to Ambulatory Patients After FDA Recommends Removal of Voluntary Hold for Ambulatory Population

The U.S. Food and Drug Administration (FDA) has announced that the agency is now recommending the removal of Sarepta Therapeutics’ voluntary hold for ambulatory patients eligible to receive ELEVIDYS. In its own press release, Sarepta…

Learn More

Update on ELEVIDYS Shipments in the U.S.

This evening, Sarepta Therapeutics issued a press release stating that it has voluntarily and temporarily paused all shipments of ELEVIDYS for Duchenne in the United States, effective close of business tomorrow, July 22, 2025. According…

Learn More

ELEVIDYS Update

Updated July 18, 2025 at 8:35 PM ET Sarepta Therapeutics has provided an update indicating that the company has received an informal request from the U.S. Food and Drug Administration (FDA) to voluntarily halt shipment…

Learn More

Post AAV Gene Therapy Resources

As therapeutic options for Duchenne advance, we are seeing continued development of additional AAV gene transfer therapies. There is currently one FDA approved gene therapy, ELEVIDYS, and several more in clinical trials. With all therapies,…

Learn More

Connecting with Experts via PPMD For You

Need direct support for yourself or your child living with dystrophinopathy? PPMD’s PPMD For You program is here to help! This personalized program lets you schedule a one-on-one meeting with a knowledgeable PPMD team member to…

Learn More

PPMD Announces Children’s Hospital of Philadelphia as CDCC, Recipient of Inaugural Clinical Research Designation

PPMD is pleased to announce the expansion of its renowned Certified Duchenne Care Center (CDCC) Program with the certification of the Neuromuscular Program at Children’s Hospital of Philadelphia (CHOP). In a milestone for the program,…

Learn More

Inclusive Summer Activities

As the school year comes to a close, we are all looking forward to a fun-filled summer! We know that planning outings and activities can sometimes feel overwhelming, especially when considering accessibility and necessary accommodations…

Learn More

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

Join Our Mailing List