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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May 27, 2025

Governor Abbott Signs Duchenne Newborn Screening into Law in Texas

We are pleased to share a significant milestone for the Duchenne community: Texas Governor Greg Abbott has officially signed Senate Bill 1044 into law! This legislation requires the Texas Department of State Health Services to…

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May 21, 2025

Florida Signs Duchenne Newborn Screening into Law

We are delighted to share exciting news for the Duchenne community: Governor Ron DeSantis has officially signed Florida’s Duchenne newborn screening legislation (HB 1089 / SB 524) into law. With this historic act, Florida becomes…

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May 12, 2025

Budget Reconciliation Update and the Impact on Medicaid

PPMD is closely monitoring the U.S. House of Representatives Energy and Commerce Committee markup of the Committee’s budget reconciliation text, scheduled for tomorrow, Tuesday, May 13, 2025, at 2:00 PM ET. Updated legislative language released…

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May 7, 2025

An Update on PPMD’s Continued Strategy for Advancing Duchenne Newborn Screening

On April 3, 2025, the U.S. Department of Health and Human Services (HHS) announced the dissolution of the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), an unexpected development that has left a…

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April 21, 2025

Watch: Duchenne Newborn Screening – Next Steps Following ACHDNC Dissolution (Webinar Recording)

Last week, PPMD and the Muscular Dystrophy Association provided insights into the current status of the Duchenne nomination to the federal Recommended Uniform Screening Panel (RUSP) for newborn screening following the dissolution of the Advisory…

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