feature

Mapping Out a Path Forward: PPMD Convenes Representatives from 21 Academic Centers and 11 Industry Partners for 2024 Cardiac Care Workshop

Last week, PPMD held the 2024 Cardiac Care Workshop in Baltimore, Maryland, which included 40 academics and professionals and representatives from 11 industry partners to discuss how to move beyond the current state of cardiac…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

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),…

Learn More

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…

Learn More

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,…

Learn More

Welcoming Jodi Wolff, PhD: A Passionate Advocate Joins PPMD’s Team as Chief Program Officer

We are thrilled to welcome the newest addition to the PPMD team, Jodi Wolff, PhD, who joins us as our Chief Program Officer! Jodi brings with her a wealth of experience and an unwavering passion…

Learn More

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…

Learn More

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…

Learn More

Join Our Mailing List