Study shows real-world benefits of onasemnogene abeparvovec in younger SMA patients

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Apr 29 / Springer Healthcare

Study shows real-world benefits of onasemnogene abeparvovec in younger SMA patients

Neuroscience Spinal Muscular Atrophy EPNS-2022

Description

Lena-Luise Becker discusses findings from a real-world study showing that spinal muscular atrophy patients younger than 8 months of age are the most likely to benefit from onasemnogene abeparvovec, while those older than 24 months have a lower likelihood of treatment response and a higher risk for side effects.

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