948. Risdiplam in Presymptomatic Spinal Muscular Atrophy.
作者: Richard S Finkel.;Laurent Servais.;Dmitry Vlodavets.;Edmar Zanoteli.;Maria Mazurkiewicz-Bełdzińska.;Yuh-Jyh Jong.;Aledie Navas-Nazario.;Mohammad Al-Muhaizea.;Alexandra P Q C Araujo.;Leslie Nelson.;Yi Wang.;Birgit Jaber.;Ksenija Gorni.;Heidemarie Kletzl.;Laura Palfreeman.;Michael Rabbia.;Dave Summers.;Eleni Gaki.;Kathryn R Wagner.;Paulo Fontoura.;Michelle A Farrar.;Enrico Bertini.; .
来源: N Engl J Med. 2025年393卷7期671-682页
Risdiplam, an oral pre-messenger RNA splicing modifier, is an efficacious treatment for persons with symptomatic spinal muscular atrophy (SMA). The safety and efficacy of risdiplam in presymptomatic disease are unclear.
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