May 8, 2024

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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May 7, 2024

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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May 6, 2024

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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May 2, 2024

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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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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