Wave Life Sciences Announces Positive Interim Data from FORWARD-53 Clinical Trial Evaluating WVE-N531 in Boys with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
Wave Life Sciences .(WVE) GlobeNewswire News Room·2024-09-24 19:30
Mean muscle content-adjusted dystrophin expression of 9.0% and unadjusted dystrophin of 5.5%, with high consistency across participants, in a prespecified analysis; dystrophin was comprised of two isoforms consistent with Becker muscular dystrophy patients who display milder disease Data demonstrated meaningful improvement in serum biomarkers for muscle health, with localization of WVEN531 in myogenic stem cells and regeneration of myofibers Skeletal muscle concentrations of ~41,000 ng/g combined with 61-da ...