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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 […]
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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 […]
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Global Nonprofit CureDuchenne to Host FUTURES National Conference for the Duchenne and Becker Muscular Dystrophy Community on May 23-26 in Orlando, FL
Four-Day Conference Will Feature Key Opinion Leaders and Notable Speakers including Peter Marks, M.D., Ph.D., […]
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WATCH: Community Progress in Duchenne Newborn Screening (Webinar Recording)
PPMD, in collaboration with the Muscular Dystrophy Association (MDA), the EveryLife Foundation for Rare Diseases, […]
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Baby Duchenne research network receives $250K from PPMD
A $250,000 award from the Parent Project Muscular Dystrophy (PPMD) will support the establishment of […]
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Rolling Through the Magic: Exploring Disneyland in a Power Wheelchair
Disney Parks can be great places for someone with a power wheelchair to enjoy the […]
