REGENXBIO GENE THERAPY
RGX-202, an investigational gene therapy for Duchenne, continues to show a favorable safety profile with no serious adverse events, no liver injury signals, and reductions in key muscle damage biomarkers one year after treatment. Interim results from seven participants indicate improved functional performance and stable cardiac function, with strong microdystrophin expression, while Phase III topline data are expected in Q2 2026 as the trial continues enrolling participants.
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