Evaluation of real-life outcome data of patients with spinal muscular atrophy treated with nusinersen in Switzerland

Anne Tscherter,Christina T Rüsch, Dominique Baumann, Cornelia Enzmann,Oswald Hasselmann,David Jacquier,Hans H Jung,Michelle E Kruijshaar,Claudia E Kuehni,Christoph Neuwirth,Georg M Stettner,Andrea Klein

Neuromuscular Disorders（2022）

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摘要

•Nusinersen is an effective treatment, especially in young children with SMA type 1.•Older patients treated with nusinersen gained or stabilised their motor function.•Infections prompt the need for ventilatory and nutritional support in SMA1 infants.•Evaluation of speech development in nusinersen early treated children is important.

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关键词

Spinal muscular atrophy,Treatment efficacy,Real-life outcome data,Switzerland,Nusinersen

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