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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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Handling Emergencies
See the below for resources to assist in a Duchenne emergency: The post Handling Emergencies […]
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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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PPMD Hosts 2024 Cardiac Workshop, May 9-10
PPMD is excited to bring together experts in the cardiac field, spanning clinicians and researchers, […]
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Paying tribute to the allied healthcare professionals in my life
Last Saturday, I attended a focus group on behalf of a local patient advocacy community […]
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REGENXBIO Reports First Quarter 2024 Update On Duchenne muscular dystrophy Program
REGENXBIO provided a clinical and regulatory update on their Duchenne muscular dystrophy program highlighting progress […]
