Regenxbio's one-time gene therapy for Duchenne.
1 report so far. New developments are added to this thread as they happen.
- Oct 25, 2026BIG TRIAL MEETS ITS MAIN GOALNo-placebo trial · 31 boys
One-time gene therapy for Duchenne muscle disease met its main trial goal
In 31 boys with Duchenne, a single infusion got muscles making a stand-in for a missing protein above a target level. Early walking and standing tests favored treatment.