October 20, 2026

Sam’s Night Celebrates 21 Years of Fighting to End Duchenne, Raises $1.7 Million for PPMD

The Killian family of Rockwall, Texas, is celebrating another successful year of Sam’s Night, their annual fundraiser benefiting Parent Project Muscular Dystrophy (PPMD), the leading U.S. nonprofit driving Duchenne and Becker muscular dystrophy care, research,…

Learn More

October 15, 2026

Care Should Grow With You: Building a Better Future for Adults With Duchenne and Becker

For families living with Duchenne and Becker, planning for adulthood should bring possibilities: education, relationships, work, greater independence, and the opportunity to build a life shaped by individual goals and choices. Too often, it also…

Learn More

October 5, 2026

Stay Healthy This Respiratory Season: What to Know and How to Prepare

As school gets underway and winter approaches, respiratory viruses like influenza (flu), RSV, and COVID-19 are more likely to spread. This year, and every year, it’s important to protect yourself, your children, and your family…

Learn More

September 30, 2026

Catch up on the 31st Congress of the World Muscle Society

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,…

Learn More

September 22, 2026

Catch up on PPMD Together: San Francisco

This past weekend, PPMD headed west for PPMD Together: San Francisco, CA, bringing together families, clinicians, industry partners, and local organizations for two days of connection, learning, and collaboration. Following an evening reception on Friday,…

Learn More

September 18, 2026

PPMD Celebrates Patient-Centered Advocacy at RAPS Convergence 2026

Earlier this week, PPMD joined global regulatory professionals, patient advocates, industry leaders, and others from across the healthcare community at RAPS Convergence 2026 in Charlotte, North Carolina. This year marked the 50th anniversary of the…

Learn More

September 8, 2026

Novartis Shares Del-Zota Regulatory Update: FDA Accepts BLA for Priority Review

Novartis announced today that the U.S. Food and Drug Administration (FDA) has notified the company that the Biologics License Application (BLA) of del-zota (delpacibart zotadirsen) is sufficiently complete to permit a substantive review. According to…

Learn More

August 24, 2026

Update on Deramiocel: PDUFA Date Extended

Today, Capricor Therapeutics formally announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s amendment to the Biologics License Application (BLA) for Deramiocel, and the FDA will be extending the current Prescription…

Learn More

August 24, 2026

Precision Biosciences Doses First Patient in FUNCTION-DMD Trial of PBGENE-DMD

Precision BioSciences, Inc. today announced the dosing of the first patient in the Phase 1/2 FUNCTION-DMD clinical trial, evaluating the safety, tolerability, and efficacy of PBGENE-DMD for the treatment of Duchenne. PBGENE-DMD is Precision’s investigational…

Learn More

August 21, 2026

Deramiocel: Where We Are Today

As we approach August 22, the FDA’s current Prescription Drug User Fee Act (PDUFA) action date for Capricor Therapeutics’ Biologics License Application (BLA) for Deramiocel, PPMD recognizes how closely the Duchenne community is watching for…

Learn More