Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold on SRP-5051 […]
Published program data shows Dyne’s FORCE™ platform achieves enhanced exon skipping and prolonged dystrophin restoration in Duchenne in vivo mdx mouse model CureDuchenne provided funding to Dyne Therapeutics and we […]
Kevin Flanigan, MD is a leading expert in the development of gene therapies for Duchenne, and the director of the Center for Gene Therapy in the Abigail Wexner Research Institute […]
Duchenne muscular dystrophy was nominated to be added to the Recommended Uniform Screening Panel (RUSP), a list of disorders that are screened for at birth. This is an important step […]
Sarepta Therapeutics Shares Clinical Data and Integrated Analysis.
Dear Duchenne Community, We are writing to provide some additional information to accompany our press release (attached) this week about the status of the rolling New Drug Application at the […]
Capricor Announces More Good News For CAP-1002
Italfarmaco released positive topline data from their Phase 3 trial in Givinostat, an anti-fibrotic for use in ambulant boys with Duchenne over 6 years old and on a chronic steroid […]
