Nusinersen in adult patients with 5q spinal muscular atrophy: A multicenter observational cohorts' study.

Juan F Vázquez-Costa,Mónica Povedano,Andrés E Nascimiento-Osorio,Antonio Moreno Escribano,Solange Kapetanovic Garcia,Raul Dominguez,Jessica M Exposito,Laura González,Carla Marco,Julita Medina Castillo,Nuria Muelas,Daniel Natera de Benito,Nancy Carolina Ñungo Garzón,Inmaculada Pitarch Castellano,Teresa Sevilla,David Hervás

medRxiv（2022）

引用 12|浏览4

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摘要

Nusinersen treatment is associated with some improvements in adult SMA patients. Most severely affected patients with complex spines are probably those with the most unfavorable risk-benefit ratio.

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关键词

adults,cohort study,nusinersen,spinal muscular atrophy,treatment

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