January 12, 2026

PPMD’s Race to End Duchenne Team Member Claims Victories in Both 5K and 10K as Community Raises Funds for Duchenne and Becker Research and Care

PPMD celebrated a powerful and inspiring Walt Disney World® Marathon Weekend as part of its Race to End Duchenne endurance fundraising program this past weekend! PPMD runners, families, and supporters came together in Orlando, Florida,…

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December 22, 2025

Watch: Capricor Therapeutics — HOPE-3 Community Update (Webinar Recording)

PPMD recently held a community webinar with Capricor Therapeutics, during which the company shared their most recent results from the Phase 3 HOPE-3 trial evaluating deramiocel (CAP-1002), Capricor’s investigational cell therapy for the treatment of…

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December 16, 2025

Historic Victory: Duchenne Added to the Recommended Uniform Screening Panel (RUSP)

Today marks a monumental victory for the Duchenne community. The U.S. Department of Health and Human Services (HHS) has officially added Duchenne to the Recommended Uniform Screening Panel (RUSP)—the national list of conditions recommended for…

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December 8, 2025

Dyne Therapeutics Announces Positive Topline Results from Phase 1/2 DELIVER Trial of Z-Rostudirsen in Duchenne

Dyne Therapeutics has announced positive topline results from its Registrational Expansion Cohort (REC) of the Phase 1/2 DELIVER trial of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) in people living with Duchenne who are amenable…

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December 5, 2025

Honoring Every Journey in Duchenne and Becker: Advancing Therapies, Access, and Care For All

For more than three decades, Parent Project Muscular Dystrophy (PPMD) has been committed to bringing therapies closer to, and improving care for, every person living with Duchenne and Becker muscular dystrophy. The Duchenne and Becker…

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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…

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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…

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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…

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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,…

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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…

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