April 30, 2024

Thank You for Honoring PPMD’s 30th Anniversary and Fighting for Every Future With Us

As I’ve reflected on the incredible journey of PPMD these past few weeks, I am overwhelmed with gratitude for each and every one of you who have supported us over three decades. From the founding…

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April 29, 2024

Catch Up on PPMD’s Inaugural PPMD Together Event in Cincinnati, Ohio

On April 19th and 20th, PPMD launched our brand new PPMD Together meeting series in Cincinnati, Ohio. The date and location held immense significance as we gathered to commemorate 30 years since the incorporation of…

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April 29, 2024

PPMD Awards $250,000 to Support Clinical Research Network for Duchenne Babies Identified by Newborn Screening

PPMD is excited to announce a $250,000 award to Bo Hoon Lee, MD, from the University of Rochester to support  the development of a clinical research network for Duchenne babies identified through newborn screening efforts…

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April 23, 2024

Duchenne UK and PPMD Award $500,000 to Evaluate Safety, Tolerability of Muscle Progenitor Cells in Phase 1 Trial

PPMD and Duchenne UK are excited to announce the recipient of their Joint Call for Therapeutic Projects. Pediatric neuromuscular neurologist Professor Peter Kang MD, from the University of Minnesota Medical School, has been awarded the…

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April 22, 2024

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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April 18, 2024

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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April 16, 2024

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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April 15, 2024

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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April 15, 2024

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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April 9, 2024

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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