As an early investor in Entrada Therapeutics, we are pleased to share their progress on their exon-skipping programs in Duchenne. For Exon 44: Entrada completed enrollment for the first cohort […]
Sarepta has completed the ESSENCE study, which is a confirmatory trial aimed at supporting full FDA approval of the exon-skipping PMOs Amondys 45 and Vyondys 53. The study did reinforce […]
Updated 10/31/2015:Letter to the community: October 30, 2025RegenXBio announced that they have completed enrollment in the Phase 1/2 portion of their RGX-202 microdystrophin gene therapy trial, with topline data expected […]
Italfarmaco issues a letter to address the recent online discussions about the individuals who have passed away while being treated with Duvyzat (givinostat), confirming that they have determined the deaths […]
CureDuchenne and Andreas S. Barth, MD, PhD, FAHA, for a community webinar about Optimizing Cardiac Outcomes in Duchenne. Watch HERE
Full-Day Conference Will Bring Education, Support, and Connection to Families and Healthcare Providers Serving the Duchenne and Becker Community Newport Beach, Calif., September 18, 2025 — CureDuchenne, a global leader […]
Limited-Edition Violent Gentlemen Hockey Club Tees Benefiting the CureDuchenne. Your purchase makes a difference.Every tee supports CureDuchenne’s mission to improve the lives of everyone affected by Duchenne muscular dystrophy—by accelerating […]
Results published in the Annals of Clinical and Translational Neurology show that long-term treatment with givinostat, a histone deacetylase (HDAC) inhibitor, in combination with corticosteroids, delayed disease progression in ambulant individuals with […]
