December 17, 2024

Your gift to PPMD is a commitment to accelerating progress

As a pediatric neurologist who takes care of children with neuromuscular diseases, I’ve had the privilege of witnessing first-hand the benefit many of our therapies can have for individuals living with Duchenne. Your belief in…

Learn More

December 17, 2024

PPMD Convenes GI, GU, and Swallowing Experts for Two-Day Workshop

Earlier this month, PPMD convened experts from across the country in Columbus, Ohio for a two-day workshop focused on the development of care standards for gastrointestinal, urological, and swallowing issues in Duchenne and Becker.  As…

Learn More

December 16, 2024

PepGen Announces Clinical Hold in the U.S. on IND Application to Initiate CONNECT2-EDO51 Phase 2 Study of PGN-EDO51

PPMD is disappointed to learn that PepGen Inc. has received a full clinical hold notice from the U.S. Food and Drug Administration (FDA) regarding its Investigational New Drug (IND) application to initiate the CONNECT2-EDO51 clinical…

Learn More

December 16, 2024

Edgewise Therapeutics Announces Positive Topline Results from CANYON Phase 2 Trial of Sevasemten in Individuals with Becker

Edgewise Therapeutics, Inc., has announced positive topline results from the double-blind, randomized, placebo-controlled Phase 2 CANYON trial of sevasemten in individuals with Becker muscular dystrophy. Sevasemten is an orally administered small molecule inhibitor designed to…

Learn More

December 11, 2024

Advance the Research Evolution: Introducing PPMD’s Dystrophinopathy Clinical Research Network

In 1984, the word “Duchenne” entered my world. The progress we have made since then is astounding. For decades, we have united in our unwavering commitment to end Duchenne, and with recent achievements in this…

Learn More

December 6, 2024

Catch up on PPMD Together: Virtual Edition – A Gathering of Families for Connection, Support, and Growth

Last month, families from around the world came together for PPMD Together: Virtual Edition. This unique virtual gathering provided families a space to connect, share their experiences, and find support from one another as they…

Learn More

November 28, 2024

This Year, I Am Grateful For Our Journey Together

As I prepare for another holiday to begin, I am again overwhelmed with gratitude for the compassionate, resilient community we’ve built through PPMD. You are each an extension of my family, the family we have…

Learn More

November 26, 2024

Equitable Access in Clinical Trials—A Call for Action and Collaboration

Living with Duchenne and Becker presents unique challenges, especially for families from underserved or marginalized communities. Health disparities, delayed diagnoses, and limited access to care and clinical research create barriers that often result in diminished…

Learn More

November 21, 2024

WATCH: RGX-202 – AFFINITY DUCHENNE Pivotal Program and Functional Data (Webinar Recording)

REGENXBIO recently joined PPMD for a community webinar to provide an update on its AFFINITY DUCHENNE trial of RGX-202, REGENXBIO’s microdystrophin gene therapy. Dr. Naz Dastgir discussed the company’s pivotal trial plans and shared interim…

Learn More

November 20, 2024

PPMD Culminates 30th Anniversary with Celebratory “Investing in Every Future” Event

PPMD commemorated its 30th anniversary with the “Investing in Every Future” celebratory event on November 14, 2024, in New York City. With some 200 attendees, this meaningful gathering honored three decades of progress and the resilience…

Learn More