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Edgewise Therapeutics Announces Positive 2-Month Interim Results From The ARCH Open-Label Study Of EDG-5506 In Adults With Becker Muscular Dystrophy (BMD)
Significant decreases in key biomarkers of muscle damage.
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 […]
Nationwide Researchers Announce Restoration of Full-Length Dystrophin in Humans
We’re thrilled to announce that researchers at Nationwide Children’s Hospital have documented the first-ever creation of full-length dystrophin in a human as a response to gene therapy. CureDuchenne has contributed […]
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 […]
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 […]
REGENXBIO AFFINITY DUCHENNE TRIAL DELAYED
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 […]
Solid Updates Strategy to Develop SGT-001 & SGT-003 for Duchenne
Today, Solid Biosciences announced a change to its corporate strategy to better align resources with goals to bring SGT-001 and SGT-003 to patients. Key Takeaways: Enrollment in IGNITE DMD, its […]
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 […]