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REGENXBIO ANNOUNCES PHASE I/II TRIAL OF RGX-202, A NOVEL GENE THERAPY CANDIDATE FOR DUCHENNE MUSCULAR DYSTROPHY, IS ACTIVE AND RECRUITING PATIENTS
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 Announces Selection of Second Clinical Trial Candidate
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 […]
Wave Life Sciences announced a positive update from the initial cohort of the Phase 1b/2a study
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.
Santhera and ReveraGen Complete NDA Submission to FDA for Vamorolone in Duchenne Muscular Dystrophy
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 […]
Edgewise Therapeutics announces six sites are open for enrollment for the Canyon trial
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 […]
PepGen Presents Data from its Duchenne Muscular Dystrophy Program at World Muscle Society Congress
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 Presents Preclinical Data from its Duchenne Muscular Dystrophy Programs Targeting Exons 51 and 53 at World Muscle Society 2022 Congress
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 […]