Dr. Kevin Flanigan and his team at the Flanigan Lab at Nationwide Children’s Hospital are hard at work researching duplication mutations. They have created a new mouse model for […]
We couldn’t be prouder that CureDuchenne has played a critical role in the development of what we hope will be the first-ever pharmaceutical treatments for Duchenne muscular dystrophy. Investments by […]
To say I have witnessed young men and boys with Duchenne face heartbreaking challenges is an understatement. Duchenne is one of the toughest diseases in the world. That’s why […]
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. […]
NEWPORT BEACH, Calif., September 1, 2015 – CureDuchenne Ventures LLC, a company that funds research to find a cure for Duchenne muscular dystrophy, announced today that they have invested $1 […]
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 […]
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has filed the New Drug Application (NDA) for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Approximately 13% of people with Duchenne muscular dystrophy are estimated to have a mutation addressable by Eteplirsen/exon 51 skipping.
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for eteplirsen, a potential treatment for patients with Duchenne Muscular Dystrophy (DMD) who are amenable to skipping exon 51. The Rare Pediatric Disease Designation supplements the Orphan Drug Designation and Fast Track Status previously granted by the FDA for eteplirsen.
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for drisapersen, a potential treatment for patients with Duchenne Muscular Dystrophy (DMD) who are amenable to exon 51 skipping treatment. The FDA has previously granted drisapersen Orphan and Fast Track Status, Breakthrough Therapy Designation, as well as Priority Review status, which is designated to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists.
