Type I spinal muscular atrophy patients treated with nusinersen: 4-year follow-up of motor, respiratory and bulbar function.

European journal of neurology（2023）

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摘要

Our results confirm the safety profile previously reported and support the durability of the efficacy of nusinersen at 4 years with an overall stability or mild improvement and no evidence of deterioration over a long period of time.

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long-term results,longitudinal study,motor function,nusinersen,spinal muscular atrophy

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