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Del-desiran for DM1 wins FDA’s breakthrough therapy designation
The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to delpacibart etedesiran […]
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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 […]
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Meeting the challenges of DMD with laughter, love, and learning
Life with Duchenne muscular dystrophy (DMD) doesn’t get easier as time goes by, but it’s […]
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Dosing paused in Pfizer DMD gene therapy trial after patient death
Dosing has been paused in a Phase 3 trial testing fordadistrogene movaparvovec in children with […]
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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 […]
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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 […]
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Update on Pfizer’s DAYLIGHT Gene Therapy Trial for Duchenne
The Duchenne muscular dystrophy community recently faced a profound loss—a young participant in Pfizer’s Phase […]
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AMO Pharma to start AMO-02 Phase 3 trial in adult-onset DM1
AMO Pharma has announced it will conduct a Phase 3 trial of AMO-02 (tideglusib), its […]
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Handling Emergencies
See the below for resources to assist in a Duchenne emergency: The post Handling Emergencies […]
