Continued benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy: 5-year update of the NURTURE study.

Thomas O Crawford,Kathryn J Swoboda,Darryl C De Vivo,Enrico Bertini,Wuh-Liang Hwu,Richard S Finkel,Janbernd Kirschner,Nancy L Kuntz,Aledie Navas Nazario,Julie A Parsons,Astrid Pechmann,Monique M Ryan,Russell J Butterfield,Haluk Topaloglu,Tawfeg Ben-Omran,Valeria A Sansone,Yuh-Jyh Jong,Francy Shu, Cong Zhu, Stephanie Raynaud, Tiffany R Lago, Angela D Paradis,Richard Foster, Russell Chin,Zdenek Berger

Muscle & nerve（2023）

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摘要

These results demonstrate the value of early treatment, durability of treatment effect, and favorable safety profile after ~5 y of nusinersen treatment. Inclusion/exclusion criteria and baseline characteristics should be considered when interpreting presymptomatic SMA trial data.

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关键词

motor function, NURTURE, nusinersen, safety, spinal muscular atrophy

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