At the core of the MAA submission are positive data from the pivotal Phase 2b VISION-DMD study which comprised a (1) 24-week period to demonstrate efficacy and safety of vamorolone […]
09/29/22 8:30 AM EDT CAMBRIDGE, Mass., Sept. 29, 2022 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has […]
Participants currently enrolled in MARINA and MARINA-OLE™ trials may continue to be treated with AOC 1001 Avidity received Investigational New Drug (IND) clearance for FSHD and DMD studies from FDA; programs […]
Edgewise Therapeutics, Inc., (NASDAQ: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for the treatment of rare muscle disorders, announced today positive 4-month interim results […]
– Initiation of LYNX Phase 2 clinical trial in individuals with DMD expected in Q4 2022 – Boulder, Colo., (September 7, 2022) – Edgewise Therapeutics, Inc., (NASDAQ: EWTX), a clinical-stage […]
As an early funder of Dyne Therapeutics, CureDuchenne is pleased to share that Dyne has dosed the first participant in their clinical trial in Duchenne amenable to skipping exon 51.
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 […]
