If you live in Texas, are amendable to exon skipping 51, and you are having difficulty accessing Exondys 51, you or your provider may file a complaint to HPM_Complaints@hhsc.state.tx.us. Exondys […]
CureDuchenne is pleased to share the BioMarin Quarterly Update to the Duchenne Community. It covers important information about next week’s FDA Advisory Committee meeting and other key updates from […]
When our son, Hawken was diagnosed with Duchenne almost 13 years ago, my husband and I felt there couldn’t be too many things worse for a family to experience. […]
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 […]
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 […]
