Duchenne muscular dystrophy was nominated to be added to the Recommended Uniform Screening Panel (RUSP), a list of disorders that are screened for at birth. This is an important step […]
Dear Duchenne Community, We are writing to provide some additional information to accompany our press release (attached) this week about the status of the rolling New Drug Application at the […]
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 […]
Italfarmaco released positive topline data from their Phase 3 trial in Givinostat, an anti-fibrotic for use in ambulant boys with Duchenne over 6 years old and on a chronic steroid […]
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 […]