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 […]
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 […]
For some people it takes a trophy or medal to denote a champion. For those in the fight against Duchenne Muscular Dystrophy, there are all kinds of champions and […]
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 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 […]
Sarepta Therapeutics released the 168 week data for their exon skipping drug, eteplirsen, this morning. During a three year period, 7-13 year old boys with Duchenne (the age range of […]
CureDuchenne is proud to have supported all types of Duchenne research over the past 10 years. That is why CureDuchenne has been a leader in funding exon skipping, stop codon, […]
Thanks to GSK, Prosensa and Sarepta and, the landscape is changing for the patients and families that are suffering from Duchenne, a severe muscle disease that shortens the life expectancy […]
CureDuchenne was honored to host a webinar today, in collaboration with PPMD and MDA to discuss accelerated approval for drugs for rare diseases. Dr. Robert Temple, director of the […]
