Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort study
2019 ◽
2020 ◽
Vol 28
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pp. 38-43
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2017 ◽
Vol 377
(18)
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pp. 1713-1722
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2021 ◽