January 2, 2025

Capricor Therapeutics Completes Submission of Biologics License Application to the U.S. FDA for Deramiocel

PPMD is excited to learn that Capricor Therapeutics has completed the submission of its rolling Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking full approval for deramiocel (CAP-1002) for the…

Learn More

December 31, 2024

Double your impact in advancing the research evolution by the end of 2024

As the clock winds down on 2024 and we prepare to ring in the new year, I am awestruck by this community’s accomplishments and full of hope for the future. We have important work yet…

Learn More

December 31, 2024

Give before midnight TONIGHT to double your impact in developing PPMD’s DCRN

Happy New Year’s Eve! We’re almost there. At midnight we will ring in a new year, one that I hope will bring with it incredible experiences, happy memories, and lots of love. I am passionate…

Learn More

December 30, 2024

Together, let’s advance the research evolution

For my fellow adults living with Duchenne and Becker, today’s reality is vastly different from the one we were diagnosed into. We are living longer lives and feeling stronger while doing so. Many of us…

Learn More

December 23, 2024

Life is about time and timing when living with Duchenne

As a family living with Duchenne, time is everything to us. Decades of trials and research have paved the way for a new era, ushering in a reality with a growing landscape of both approved…

Learn More

December 19, 2024

WATCH: Sevasemten – CANYON Trial Topline Results (Webinar Recording)

Edgewise Therapeutics recently joined PPMD for a breaking news community webinar, during which Edgewise provided an update on the recently released topline results from its CANYON double-blind, randomized, placebo-controlled design to investigate the effect of…

Learn More

December 19, 2024

Support PPMD’s commitment to moving the science further, faster

Greetings from Indiana! I write today letting you know that thanks to a generous pledge from a local family I know well, a $100,000 matching gift will be made to PPMD. That means every dollar…

Learn More

December 18, 2024

Percheron Announces Termination of Phase 2B Study of Avicursen for Duchenne

PPMD is disappointed to learn of Percheron Therapeutics’ recent decision to terminate the company’s ongoing Phase 2b randomized, placebo-controlled trial of avicursen (ATL1102) in non-ambulatory individuals living with Duchenne following the release of topline six-month…

Learn More

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