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Sarepta Clinical Trials Community Bulletin

Sarepta Therapeutics is currently running multiple clinical trials in a few different countries. They are at various stages and for a range of ages. Sarepta issued an update for the […]

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Pfizer CIFFREO Webinar

Pfizer’s Global Clinical Lead for Rare Neurological Diseases, Beth Belluscio, MD-PhD, and Debra Miller, Founder and CEO of CureDuchenne, sat down to educate the Duchenne community on Pfizer’s CIFFREO Trial […]

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Entrada Presents New Data

Entrada Therapeutics announced encouraging new data on their exon 44 skipping program following their presentation at TIDES USA 2022. CureDuchenne provided funding to Entrada last year as part of their Series […]


Initiation of AFFINITY DUCHENNE™ trial of RGX-202 for the treatment of Duchenne will be delayed. This January, we shared the news that REGENXBIO received clearance from the U.S. FDA and would […]

Pfizer CIFFREO Clinical Hold Lifted

Pfizer’s Letter to the Duchenne Community We are pleased to share the good news that several regulatory authorities have approved the re-start of our Phase 3 ambulatory trial (CIFFREO) for […]

Edgewise Begins New Observational Trial

We are pleased to share that Edgewise Therapeutics is sponsoring a natural history study in Becker muscular dystrophy, to better understand the course of disease and define endpoints to be […]