Onasemnogene abeparvovec well tolerated in UK SMA cohort

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May 01 / Springer Healthcare

Onasemnogene abeparvovec well tolerated in UK SMA cohort

Neuroscience Spinal Muscular Atrophy EPNS-2022

Description

Vasantha Gowda describes the outcomes of a UK cohort of children with spinal muscular atrophy given onasemnogene abeparvovec, noting that the gene therapy was well tolerated in children aged from 2 months to 3 years and 10 months.

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