Blog

Ohio to Begin Screening Newborns for Duchenne

PPMD is excited to announce another significant milestone in Duchenne newborn screening: Ohio will be the first state to screen all babies for Duchenne! More than 129,000 babies are born in Ohio every year, and…

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PPMD Celebrates 30th Anniversary with Launch of PPMD Together Meeting Series in Cincinnati, Ohio

PPMD is excited to mark its 30th anniversary at the launch of the PPMD Together meeting series, taking place April 19-20, 2024, in Cincinnati, Ohio. Founded three decades ago by a small group of passionate parents and grandparents…

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Edgewise Therapeutics Announces Positive Two-Year Topline Results from ARCH Study of EDG-5506

Edgewise Therapeutics, Inc., has announced positive two-year topline results from the ARCH open label study of sevasemten (EDG-5506) in adults with Becker. Sevasemten is an orally administered small molecule inhibitor designed to prevent contraction-induced muscle…

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Duchenne Siblings Study Published: Assessing Early Diagnosis from the Perspective of Parents with Multiple Children Diagnosed with Duchenne

For more than a decade, PPMD has been at the forefront of advocating for Duchenne newborn screening. In 2023, we expanded our ongoing work in the space by conducting a parent survey to build evidence…

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PPMD Launches First State-Specific Duchenne Advocacy Day in Illinois

PPMD is proud to announce the launch of our first state-specific Duchenne Advocacy Day in Illinois! This initiative marks a significant milestone in PPMD’s efforts to advance legislative progress for Duchenne at the state level….

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60+ Experts Unite to Author Consensus Statement Championing Duchenne Newborn Screening

Over 60 leading experts in genetics, pediatrics, neurology, and public health advocates have authored a consensus statement in support of implementing Duchenne newborn screening. Key points of the consensus statement include: Timely Intervention: Early screening…

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WATCH: Community Update with ITF Therapeutics – Introducing DUVYZAT™ (givinostat) (Webinar Recording)

ITF Therapeutics recently joined PPMD for a webinar to provide the Duchenne community with an opportunity to meet members of the ITF Therapeutics team to learn more about their organization and next steps to support…

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Fiscal Year 2024 Appropriations Package Signed Into Law, Including Critical Funding for Duchenne and Becker

At the beginning of March, PPMD’s Advocacy Conference brought together more than 120 advocates from 26 different states in Washington, D.C. to advocate on behalf of the Duchenne and Becker community. This past weekend, the…

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ITF Therapeutics Shares Update on DUVYZAT™ Following FDA Approval

Following the FDA’s approval of DUVYZAT™ (givinostat) on March 21, 2024, ITF Therapeutics, LLC, the company responsible for producing and marketing DUVYZAT™ in the United States, today shared an update on the drug and related…

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FDA Grants Approval to DUVYZAT™ (Givinostat)

PPMD is excited to learn that the FDA has granted approval to DUVYZAT™ (givinostat), a histone deacetylase (HDAC) inhibitor indicated for individuals diagnosed with Duchenne muscular dystrophy from six years of age and older. DUVYZAT™…

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