This week the FDA accepted Sarepta Therapeutics’ New Drug Application for eteplirsen with priority review. This follows on the June FDA’s acceptance of BioMarin’s NDA for drisapersen, also with […]
FDA Accepts NDA for Drisapersen, the First-Ever Treatment for Duchenne Muscular Dystrophy to Reach this Milestone in the U.S., and Sarepta Therapeutics Completes NDA Submission to FDA for Eteplirsen […]
There has been a lot of recent Duchenne research news lately. As a community, we are glad to see a variety of research programs moving forward with positive results. […]
The Duchenne community is celebrating another promising step toward an approved treatment. On May 19 Sarepta Therapeutics announced plans to submit a rolling NDA for eteplirsen with the final […]
As I type this, my heart is filled with hope. A horizon that we could barely see 12 years ago has arrived. When our son Hawken was diagnosed with Duchenne […]
CureDuchenne Cares is pleased to share important information about steroids use for Duchenne patients. Use of corticosteroids is part of the standards of care for Duchenne patients. Chronic use […]
CureDuchenne is pleased to share the following communication update from BioMarin to the Duchenne community regarding developments of their exon skipping program for exon 51. CureDuchenne has provided extensive support […]
CureDuchenne is pleased to share the following update to the Duchenne muscular dystrophy community from Lilly regarding the ongoing Phase 3 trial of tadalafil in boys with Duchenne. Eli Lilly […]
Today, CureDuchenne announced a $1 million investment in Capricor Therapeutics for a clinical trial to test its CDC (adult) stem cells as a cardiac therapy for Duchenne patients. CureDuchenne’s mission […]
