GNT0004, an experimental gene therapy for Duchenne muscular dystrophy (DMD), appears to be working as intended in the initial…
Articles by Marisa Wexler, MS
Regenxbio has initiated the pivotal phase of a clinical trial testing its experimental gene therapy RGX-202 in boys…
The U.S. Food and Drug Administration (FDA) has given Atamyo Therapeutics the go-ahead to start a Phase 1b clinical…
A new patient group is being enrolled in a Phase 1/2 trial of delpacibart braxlosiran (del-brax), an experimental and potentially…
CureDuchenne is partnering with Blizzard Entertainment, maker of the massively multiplayer online role-playing game World of Warcraft, on a fundraiser…
The Committee for Medicinal Products for Human Use (CHMP) — part of the European Medicines Agency — again maintained its…
Treatment with antimiRs, an RNA-based therapy approach, was able to correct molecular defects in a cell model of myotonic…
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to SAT-3247, an oral treatment candidate for…
Duvyzat (givinostat), a newly approved treatment for Duchenne muscular dystrophy (DMD), is now available in the U.S., according…
Most children with Duchenne muscular dystrophy (DMD) start experiencing notable issues with arm function and breathing ability before they…