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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 (del-desiran, previously called AOC 1001), an investigational treatment for myotonic dystrophy type 1 (DM1) that’s about to enter Phase 3 clinical testing. The FDA gives the designation to experimental therapies that have the potential to fill unmet medical needs in…
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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 academics and professionals and representatives from 11 industry partners to discuss how to move beyond the current state of cardiac care to accelerate research, advance standards of care, and better understand Duchenne and Becker in a rapidly evolving and complicated…
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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 made better by laughter, love, and everything that’s learned along the way. Three of my seven children have DMD: Max, 18; Rowen, 15; and Charlie, 13. As you might imagine, my life is busy and challenging. Recently, Max said someone…
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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 Duchenne muscular dystrophy (DMD) following the sudden death of a boy who had been previously treated with the gene therapy, its developer Pfizer announced in a letter to the community. Pfizer indicated it does not have complete information about the…
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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 Furlong, has been named the 2024 recipient of the prestigious Sonia Skarlatos Public Service Award by the American Society of Gene & Cell Therapy (ASGCT). The award was presented during ASGCT’s Annual Meeting in Baltimore, Maryland. This esteemed award recognizes…
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Monday Family
We are blessed that our son Momo joined our family through adoption. It was a long road to bring him home that led us to understand that God’s plans are much more than we can ever imagine. Throughout our 4 year adoption journey to bring Momo home, there was another little boy waiting for a…
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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 newborn screening. These meetings serve as opportunities for the committee to discuss potential recommendations to the Secretary of the U.S.…
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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 2 DAYLIGHT study (for boys 2 years to less than 4 years of age) passed away due to cardiac arrest, over a year after receiving an investigational micro-dystrophin gene therapy aimed at treating Duchenne. At CureDuchenne, we are devastated to…
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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 investigational oral therapy for adult-onset myotonic dystrophy type 1 (DM1). The decision follows a recent meeting with the U.S. Food and Drug Administration (FDA) to review data from the Phase 2/3 REACH-CDM trial (NCT03692312). As reviewed by the agency, trial…
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Handling Emergencies
See the below for resources to assist in a Duchenne emergency: The post Handling Emergencies appeared first on CureDuchenne.
