July 11, 2025

Post AAV Gene Therapy Resources

As therapeutic options for Duchenne advance, we are seeing continued development of additional AAV gene transfer therapies. There is currently one FDA approved gene therapy, ELEVIDYS, and several more in clinical trials.  With all therapies,…

Learn More

July 11, 2025

Capricor Therapeutics Receives Complete Response Letter Regarding Deramiocel (CAP-1002)

On July 9th, the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for Capricor’s Biologics License Application (BLA) for Deramiocel (CAP-1002), an investigational cell therapy for Duchenne cardiomyopathy. This decision means…

Learn More

July 10, 2025

Connecting with Experts via PPMD For You

Need direct support for yourself or your child living with dystrophinopathy? PPMD’s PPMD For You program is here to help! This personalized program lets you schedule a one-on-one meeting with a knowledgeable PPMD team member to…

Learn More

July 7, 2025

Medicaid, Reconciliation Passage, and What Comes Next for Our Community

This past week, the federal reconciliation package officially passed. The legislation covers a broad range of priorities, but for the Duchenne and Becker community, one area stands out: Medicaid. Medicaid remains one of the most…

Learn More

July 2, 2025

Webinar Recording: Edgewise Therapeutics – Update on Sevasemten Program in Becker and Duchenne

Edgewise Therapeutics recently joined PPMD for a community webinar for an update on the company’s sevasemten program in Duchenne and Becker. The Edgewise team reviewed new Open-label Extension MESA data in Becker, as well as…

Learn More

June 30, 2025

Arizona Becomes the Latest State to Require Newborn Screening for Duchenne

We are proud to share some important news for our community: Arizona has officially joined the growing list of states requiring newborn screening for Duchenne! Thanks to the tireless efforts of passionate advocates, dedicated families,…

Learn More

June 26, 2025

Edgewise Therapeutics Shares Positive Results on Sevasemten Program for Becker and Duchenne

Edgewise Therapeutics, Inc., has announced positive results in the company’s sevasemten program for both Becker and Duchenne. These data include results from: MESA, an open label extension trial of participants with Becker who were previously…

Learn More

June 24, 2025

Capricor Shares Regulatory Update on Deramiocel BLA for Duchenne

Capricor Therapeutics has shared a regulatory update related to its Biologics License Application (BLA) for Deramiocel, the company’s novel cell therapy for the treatment of Duchenne-associated cardiomyopathy. As part of the FDA’s ongoing review, Capricor…

Learn More

June 15, 2025

A Safety Update on ELEVIDYS – June 2025

We are heartbroken to learn of the loss of another member of our community following treatment with ELEVIDYS. According to Sarepta, this represents the second reported case of acute liver failure in a non-ambulatory patient…

Learn More

June 12, 2025

PPMD Announces Children’s Hospital of Philadelphia as CDCC, Recipient of Inaugural Clinical Research Designation

PPMD is pleased to announce the expansion of its renowned Certified Duchenne Care Center (CDCC) Program with the certification of the Neuromuscular Program at Children’s Hospital of Philadelphia (CHOP). In a milestone for the program,…

Learn More