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Capricor Announces More Good News For CAP-1002
Capricor Announces More Good News For CAP-1002
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 […]
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 […]
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 […]
NS Pharma Study Shows Exon 44 Skipping Drug Efficacy
National Center of Neurology and Psychiatry and Nippon Shinyaku announce positive results for a Phase I/II study evaluating the efficacy and safety of their exon 44 skipping drug (NS-089/NCNP-02) Results […]