February 16, 2024

FDA Accepts Efficacy Supplement to Expand ELEVIDYS Indication

PPMD is excited to learn that the U.S. Food and Drug Administration (FDA) has accepted and filed Sarepta Therapeutics’ efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS. The efficacy supplement aims to ​​expand…

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February 1, 2024

Duchenne Newborn Screening Bill Passes Arizona Senate

Earlier this week, Duchenne parent and PPMD Advocate Jill Castle testified before the Arizona State Senate Health & Human Services Committee in support of Senate Bill 1020, which seeks to add Duchenne to Arizona’s newborn…

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January 26, 2024

Duchenne Added to Minnesota’s Newborn Screening Panel

PPMD is excited to announce another significant milestone: Minnesota has officially approved the addition of Duchenne to the state’s newborn screening panel! Approximately 63,000 babies are born in Minnesota every year, which means that we…

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January 25, 2024

CHMP Issues Negative Opinion for Renewal of Conditional Marketing Authorization for Translarna™ (ataluren) in Europe

PPMD is disappointed to learn that the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) today issued a negative opinion following the re-examination procedure for the conditional marketing authorization of Translarna™…

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December 21, 2023

PPMD & Community Partners Urge Timely, Transparent FDA Review of EMBARK Data

Today, PPMD joined the Best Day Ever Foundation, CureDuchenne, the Jett Foundation, the Little Hercules Foundation, The Muscular Dystrophy Family Foundation, and Team Joseph in sending a letter to the Federal Drug Administration (FDA) regarding…

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December 11, 2023

Register Now for PPMD’s 2024 Advocacy Conference

Join us in Washington, DC, March 3–5, 2024, as we continue to fight for every future. Each year, PPMD ensures that the Duchenne community is at the forefront of the hearts and minds of Congressional…

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November 29, 2023

United Nations Officially Designates September 7 as World Duchenne Awareness Day

The United Nations has marked a momentous milestone for the global rare disease community by officially designating September 7th as World Duchenne Awareness Day, set to be observed annually from 2024. The adoption of the…

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November 16, 2023

PPMD Urges UN Support for ‘World Duchenne Awareness Day’ Resolution Ahead of UN General Assembly Meeting

PPMD CEO and President Pat Furlong and Buddy Cassidy spoke yesterday in front of 60+ diplomats to encourage ambassadors to the United Nations and members of the World Health Organization to support a draft resolution…

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October 31, 2023

WATCH: Advocating for Access – Overview of Medicaid DUR and P&T Meetings (Webinar Recording)

During October’s Advocating for Access webinar, the PPMD and Little Hercules Foundation teams dug into what happens after a drug is approved with regard to how state Drug Utilization Review (DUR) boards assess how a…

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October 25, 2023

Duchenne Added to New York State’s Newborn Screening Panel

For more than a decade, PPMD has worked towards newborn screening (NBS) for Duchenne, including initiating a pilot study in New York State and developing a nomination package for federal review as a recommended condition…

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