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FierceBiotech 2026年5月14日

Regenxbio reaps pivotal win for Duchenne muscular dystrophy gene therapy

Regenxbio’s gene therapy for Duchenne muscular dystrophy has smashed the primary endpoint of its pivotal trial, securing a win for a biotech that has struggled...

Regenxbio’s gene therapy for Duchenne muscular dystrophy has smashed the primary endpoint of its pivotal trial, securing a win for a biotech that has struggled in recent months under the weight of a rejection and clinical hold from the FDA.