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Regenxbio's one-time gene therapy for Duchenne.

1 report so far. New developments are added to this thread as they happen.

  1. Oct 25, 2026
    BIG 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.