Blog

Sarepta Therapeutics Announces Update on ELEVIDYS Label, Company Restructuring

Yesterday afternoon, Sarepta issued a press release and community letter which included updates related to the non-ambulatory label for ELEVIDYS. While ELEVIDYS continues to be available for individuals living with Duchenne who are ambulant, Sarepta…

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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,…

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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…

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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…

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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…

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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…

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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,…

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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…

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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…

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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…

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