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机译:Therapeutic interventions for spinal muscular atrophy: preclinical and early clinical development opportunities
Univ Oxford, MDUK Neuromuscular Ctr, Dept Paediat, Oxford, England;
UCL, Dubowitz Neuromuscular Ctr, NIHR Great Ormond St Hosp Biomed Res Ctr, Great Ormond St Inst;
Neuromuscular Reference Ctr Dis, Dept Paediat, Liege, BelgiumMcGill Univ, Dept Pediat & Neurol, Montreal, PQ, Canada;
Antimyostatin; drug repositioning; gene therapy; smn1 gene; spinal muscular atrophy; nusinersen; risdiplam; onasemnogene abeparvovec; clinical trials;