Dyne Therapeutics, who received early funding from CureDuchenne, has received FDA Orphan Drug and Rare Pediatric Designations for DYNE-251 for the Treatment of Duchenne Muscular Dystrophy.
CureDuchenne was the original funder of cTAP. We congratulate the consortium and other authors on their recent publication looking at large data sources and investigating effects of different genotypes on […]
Myosana Therapeutics, a biotech company based in Seattle, WA, closed a new $5 million financing round to support further development of their non-viral gene therapy technology for the treatment of [...]
REGENXBIO is recruiting participants aged 4-11 years old for their microdystrophin gene therapy trial, as well as participants aged 0-11 years for an observational study to understand the prevalence of […]
Entrada Therapeutics announced the selection of a second clinical candidate, ENTR-601-45, for the potential treatment of people living with Duchenne who are exon 45 skipping amenable. Read more in the […]
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