A pooled analysis across four clinical trials in Duchenne muscular dystrophy reported a manageable cardiac safety profile over up to five years for delandistrogene moxeparvovec. The dataset focuses on longer follow-up, a critical parameter for assessing delayed toxicities from adeno-associated virus-based gene therapies. The finding supports ongoing development and continued cardiac monitoring as part of post-dosing surveillance. For the broader gene-therapy sector, the report provides further evidence that long-term safety characterization remains central to translating transformational one-time therapies into durable clinical practice.
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