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…

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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…

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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…

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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…

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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…

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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…

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November 18, 2024

REGENXBIO Initiates Pivotal Phase of AFFINITY DUCHENNE® Trial of RGX-202 Gene Therapy, Reports Positive Functional Data

REGENXBIO Inc. has announced that the AFFINITY DUCHENNE® open-label trial of RGX-202 has advanced to pivotal stage and dosed its first patient. The company also announced new, positive efficacy and safety data from the Phase…

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November 12, 2024

Calling All Adults with Duchenne & Becker: Join the 2025 PPMD Adult Advisory Committee

Have you been looking for a way to engage with the community more? Do you have a personal experience that you would like to share with the larger community or through federal and state advocacy…

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November 7, 2024

REGENXBIO Shares Updates on Phase I/II AFFINITY DUCHENNE® trial of RGX-202

Yesterday, REGENXBIO shared important updates on their Phase I/II AFFINITY DUCHENNE® trial of RGX-202 for Duchenne during their third quarter financial results update. RGX-202 utilizes a novel adeno-associated virus (AAV8) to transport a shortened version…

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November 6, 2024

Sarepta Announces Discontinuation of SRP-5051 Development for Duchenne

Sarepta Therapeutics today announced that the company is discontinuing its SRP-5051 (vesleteplirsen) development program, including the global, Phase 2, multi-arm, ascending dose MOMENTUM study. Vesleteplirsen is an investigational, next-generation treatment utilizing Sarepta’s PPMO chemistry and…

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