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Delandistrogene moxeparvovec: gene therapy for Duchenne muscular dystrophy

edpract · 2026-04-24 · canonical JSON source

2 visible annotations · policy: published · automated confidence ≥ 75.00%

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Duchenne muscular dystrophy (DMD) is a severe, progressive X-linked disorder caused by pathogenic variants in the DMD gene, resulting in the absence or markedly reduced dystrophin.1 Loss of dystrophin destabilises the sarcolemma, leading to progressive muscle damage and weakness.2 Patients typically present in early childhood with delayed motor milestones and elevated creatine kinase, progressing to loss of ambulation and premature cardiorespiratory failure.3