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 […]
Exon duplication mutations have been an area of great interest to CureDuchenne, and last year, CureDuchenne funded research specifically directed toward these mutations in Duchenne. Dr. Kevin Flanigan at Nationwide […]
Following the long awaited news of Sarepta’s phase IIB results for eteplirsen and continued Phase III progress of GSK/Prosensa’s drisapersen, as well as the recent developments with PTC Therapeutics and […]
