October 9, 2025

Catch Up on World Muscle Society 2025

Each year, the World Muscle Society (WMS) Annual Congress brings together the best minds in neuromuscular research, clinical care, patient advocacy, and industry. The meeting offers a dynamic platform for sharing new data, fostering collaborations,…

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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 11, 2025

Duchenne on the World Stage: World Duchenne Awareness Day at the United Nations

On Friday, September 5th, 2025 I had the honor of speaking at the United Nations to raise awareness and turn hope into action for World Duchenne Awareness Day.  The Permanent Representative of the State of…

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September 10, 2025

Avidity Biosciences Announces Positive New Data from EXPLORE44® and EXPLORE44-OLE™ Phase 1/2 Studies

Today, Avidity Biosciences announced new data from its EXPLORE44® and EXPLORE44-OLE™ Phase 1/2 studies of del-zota in people living with Duchenne muscular dystrophy amenable to exon 44 skipping. Del-zota is designed to deliver phosphorodiamidate morpholino…

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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 18, 2025

Expanding Leadership, Accelerating Progress

Dear PPMD Community, For more than thirty years, I have had the honor of standing beside you—parents, families, clinicians, researchers, and advocates—united in a single purpose: to end Duchenne and Becker muscular dystrophy, and to…

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