October 10, 2025

Standing Together for Access: Ensuring Duchenne Patients Can Benefit from Approved Therapies

PPMD has always fought to ensure that every individual living with Duchenne has access to safe, effective, FDA-approved treatments. This week, PPMD joined six partner organizations: Best Day Ever Foundation, Charlie’s Cure, CureDuchenne, Little Hercules…

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September 5, 2025

PPMD Kicks Off World Duchenne Awareness Day with Bad Shirt Friday, Special United Nations Documentary Premiere

PPMD will join forces with fellow Duchenne-specific organizations from around the world to celebrate World Duchenne Awareness Day on September 7. This global initiative, spearheaded by the World Duchenne Organization, aims to raise awareness about Duchenne and…

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August 14, 2025

Make Your Voice Heard: Support Adding Duchenne to the RUSP

Every year, thousands of newborns in the U.S. are screened for serious conditions whose early detection can change the course of their lives. Right now, we have a chance to ensure Duchenne muscular dystrophy is…

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August 1, 2025

Watch: Capricor Therapeutics – Regulatory Update & Clinical Insights on Deramiocel for Duchenne Cardiomyopathy

Capricor Therapeutics recently joined PPMD for a community webinar on Tuesday, July 29, 2025 to discuss the current status of Capricor’s Biologics License Application (BLA) for Deramiocel (CAP-1002). We discussed the regulatory implications of a…

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July 29, 2025

Sarepta to Resume ELEVIDYS Shipments to Ambulatory Patients After FDA Recommends Removal of Voluntary Hold for Ambulatory Population

The U.S. Food and Drug Administration (FDA) has announced that the agency is now recommending the removal of Sarepta Therapeutics’ voluntary hold for ambulatory patients eligible to receive ELEVIDYS. In its own press release, Sarepta…

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July 18, 2025

ELEVIDYS Update

Updated July 18, 2025 at 8:35 PM ET Sarepta Therapeutics has provided an update indicating that the company has received an informal request from the U.S. Food and Drug Administration (FDA) to voluntarily halt shipment…

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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…

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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…

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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,…

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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…

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