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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October 8, 2025

Building Protection: Vaccines and Dystrophinopathy

The PPMD team was recently joined by three clinical experts, Dr. Anne Pham-Huy (Pediatric Infectious Diseases Specialist, Clinical Immunologist, and Chair of Immunize Canada), Dr. Jane Taylor (Pediatric Pulmonologist at UPMC Children’s Hospital of Pittsburgh),…

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September 25, 2025

Catch Up on PPMD Together: Charlotte

This past weekend, PPMD held its last in person PPMD Together event of 2025 in Charlotte, North Carolina. Over two days, families, individuals with Duchenne and Becker muscular dystrophy, clinicians, researchers, and industry partners gathered…

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September 19, 2025

Ready for Respiratory Season: Keeping You and Your Family Healthy

With the start of school and the approach of winter, respiratory viruses are expected to rise. This year, and every year, it’s important to protect yourself, your children, and your family from respiratory infections. This…

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September 5, 2025

PPMD Kicks Off World Duchenne Awareness Day with Bad Shirt Friday, Special United Nations Documentary Premiere

PPMD will join forces with fellow Duchenne-specific organizations from around the world to celebrate World Duchenne Awareness Day on September 7. This global initiative, spearheaded by the World Duchenne Organization, aims to raise awareness about Duchenne and…

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September 4, 2025

Understanding BiPAP Webinar: Why It’s Needed, What Gets in the Way, and, How to Increase Compliance

The PPMD team was recently joined by pulmonologist Dr. Janaki Paskaradevan, psychologist Dr. Allison Clarke, and Maria Gonzalez, MSN, APRN, FNP-BC, CPN, from the Division of Pulmonary and Sleep Medicine at Ann & Robert H….

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August 27, 2025

Newly Published Consensus Guidelines Aim to Ensure Safe and Equitable Delivery of Gene Therapy in Duchenne

PPMD, in collaboration with the Muscular Dystrophy Association (MDA), is proud to share the release of newly published consensus guidelines for the delivery and monitoring of gene therapy in Duchenne muscular dystrophy (Duchenne). Developed in…

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August 14, 2025

Make Your Voice Heard: Support Adding Duchenne to the RUSP

Every year, thousands of newborns in the U.S. are screened for serious conditions whose early detection can change the course of their lives. Right now, we have a chance to ensure Duchenne muscular dystrophy is…

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July 29, 2025

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…

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July 22, 2025

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…

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