December 3, 2025

Capricor Therapeutics Announces Positive Topline Results from HOPE-3 Study of Deramiocel

Capricor Therapeutics today announced positive topline results from its pivotal Phase 3 HOPE-3 clinical trial evaluating Deramiocel, the company’s investigational cell therapy for the treatment of Duchenne muscular dystrophy. According to Capricor, the study met…

Learn More

November 27, 2025

A Thanksgiving Message from Pat

This Thanksgiving, I find myself reflecting deeply on gratitude, especially for this incredible community that feels like family. This season always brings a moment to pause, to take stock of where we’ve been and where…

Learn More

November 26, 2025

Watch the Recording: Avidity Biosciences – EXPLORE44-OLE® Topline and Functional Data Presentation

Avidity Biosciences joined PPMD for a community webinar on November 19, 2025, during which the company shared topline and functional data from their EXPLORE44-OLE® clinical trial. Avidity’s investigational therapy, delpacibart zotadirsen (abbreviated as del-zota), is…

Learn More

November 25, 2025

PPMD Launches New Study to Understand Patient and Caregiver Treatment Preferences

PPMD has a long history of working alongside our community to document treatment preferences and share those insights with regulatory authorities such as the FDA. Through town halls, listening sessions, and conversations at community events,…

Learn More

November 25, 2025

Sarepta Announces FDA Approval to Begin ENDEAVOR Cohort 8 Dosing in Non-Ambulatory Individuals Living with Duchenne

Sarepta Therapeutics, Inc. has shared that the U.S. Food and Drug Administration (FDA) has approved dosing in Cohort 8 of ENDEAVOR, an open-label, Phase 1b study assessing the expression and safety of ELEVIDYS in multiple…

Learn More

November 20, 2025

Closing Out the Year with Connection & Community at PPMD Together

Recently, the Duchenne and Becker community gathered virtually for the final PPMD Together meeting of the year. This intimate session centered on fostering friendships for everyone in the family while living with dystrophinopathy.  The meeting,…

Learn More

November 19, 2025

Avidity Biosciences Announces U.S. Managed Access Program for del-zota

Avidity Biosciences, Inc. has announced its Managed Access Program (MAP) for delpacibart zotadirsen (del-zota) for eligible individuals living with Duchenne who are amenable to exon 44 skipping in the United States. MAPs allow a limited…

Learn More

November 18, 2025

Becker Muscular Dystrophy Series – Part 2: Cardiopulmonary Care

The second session in PPMD’s Becker Muscular Dystrophy webinar series focused on cardiopulmonary care. Our clinical experts reviewed what every person living with Becker and their families should know about care for the heart and…

Learn More

November 14, 2025

FDA Approves New Safety Warning and Revised Indication for ELEVIDYS

Today the FDA announced a significant update to the labeling and indication of ELEVIDYS for the treatment of individuals living with Duchenne following reports of death based on acute liver failure (ALF) in non-ambulatory patients…

Learn More

November 10, 2025

Tips for Smooth Holiday Travel

The holidays are a special time to connect with family and friends, and for many, that involves travel. To help make your journey a little easier, PPMD has gathered some helpful travel tips and resources!…

Learn More