Blog

PPMD’s Pat Furlong Honored with ASGCT’s Sonia Skarlatos Public Service Award

PPMD is proud to share some exciting news: our very own President and CEO, Pat Furlong, has been named the 2024 recipient of the prestigious Sonia Skarlatos Public Service Award by the American Society of…

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Updates from the ACHDNC Meeting and Next Steps for Duchenne Newborn Screening

Members of the PPMD community provided testimony today during the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) quarterly meeting, voicing support for adding Duchenne to the Recommended Uniform Screening Panel (RUSP) for…

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PPMD Hosts 2024 Cardiac Workshop, May 9-10

PPMD is excited to bring together experts in the cardiac field, spanning clinicians and researchers, to the third annual PPMD Cardiac Workshop, May 9-10, 2024 to delve into current and emerging issues in cardiac care…

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Update on Pfizer’s Phase 2 Gene Therapy Trial for Duchenne

We are devastated to learn of the tragic loss of a young boy that was participating in Pfizer’s Phase 2 DAYLIGHT study. This study is evaluating the safety and dystrophin expression of fordadistrogene movaparvovec (PF-06939926),…

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WATCH: Community Progress in Duchenne Newborn Screening (Webinar Recording)

PPMD, in collaboration with the Muscular Dystrophy Association (MDA), the EveryLife Foundation for Rare Diseases, and the Little Hercules Foundation (LHF), recently hosted a webinar on the background, history, and next steps for Duchenne Newborn…

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Comprehensive Genetic Counseling Resource for Dystrophinopathies Published: A Collaborative Effort by PPMD and Expert Genetic Counselors

PPMD is pleased to share that a new genetic counseling practice resource for dystrophinopathies, including Duchenne and Becker muscular dystrophy and carriers, has been published in the Journal of Genetic Counseling. PPMD Certified Genetic Counselors,…

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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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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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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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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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