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S11 Long-term follow-up of the phase 1 START trial of onasemnogene abeparvovec gene therapy in spinal muscular atrophy type 1
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Mendell J, Shell R, Lehman K, et al
S11 Long-term follow-up of the phase 1 START trial of onasemnogene abeparvovec gene therapy in spinal muscular atrophy type 1