In December 2022, Wave Life Sciences announced a positive update from the initial cohort of the Phase 1b/2a study of WVE-N531 in three boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping.
Pratteln, Switzerland, and Rockville, MD, USA, October 27, 2022 – Santhera Pharmaceuticals (SIX: SANN) and ReveraGen BioPharma, Inc announce that they have completed the rolling submission of a new drug […]
We are pleased to announce that six sites are open for enrollment for the Canyon trial for individuals living with Becker Muscular Dystrophy! Canyon is a Phase 2 trial of […]
See full PRESS RELEASE: https://www.globenewswire.com/news-release/2022/10/13/2534198/0/en/PepGen-Presents-Data-from-its-Duchenne-Muscular-Dystrophy-Program-at-World-Muscle-Society-Congress.html
Dyne Therapeutics, who received early funding from CureDuchenne, presented promising preclinical data from their Duchenne Exon 51 and 53 programs at the World Muscle Society Congress. This was Dyne’s first […]
Edgewise Therapeutics, which received early funding from CureDuchenne, announced positive 6-month interim results from the ongoing ARCH study, an open label study of EDG-5506 in adults with Becker muscular dystrophy. Edgewise […]
CureDuchenne, as an early investor in Avidity Biosciences, is excited to share the announcement of the Phase 1/2 EXPLORE44™ clinical trial of AOC 1044 in healthy volunteers and participants with […]
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 […]
