We are pleased to announce that six sites are open for enrollment for the Canyon trial for individuals living with Becker Muscular Dystrophy! Canyon is a Phase 2 trial of […]
CureDuchenne, as an early investor in Avidity Biosciences, is excited to share the announcement of the Phase 1/2 EXPLORE44™ clinical trial of AOC 1044 in healthy volunteers and participants with […]
At the core of the MAA submission are positive data from the pivotal Phase 2b VISION-DMD study which comprised a (1) 24-week period to demonstrate efficacy and safety of vamorolone […]
CureDuchenne provided early and significant funding to Capricor Therapeutics for their Duchenne program, and we are pleased to share their latest updates. One-year results from their HOPE-2 open label extension […]
Significant decreases in key biomarkers of muscle damage.
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 […]
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’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 […]
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 […]
