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JEWELFISH: 24-Month Results from an Open-Label Study in Non-Treatment-naïve Patients with SMA Receiving Treatment with Risdiplam

Claudia A. Chiriboga,Claudio Bruno,Tina Duong,Dirk Fischer,Eugenio Mercuri,Janbernd Kirschner,Anna Kostera-Pruszczyk,Birgit Jaber,Ksenija Gorni,Heidemarie Kletzl,Imogen Carruthers,Carmen Martin,Renata S. Scalco,Paulo Fontoura,Francesco Muntoni

Journal of Neurology（2024）

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关键词
Spinal muscular atrophy,Risdiplam,SMA,Safety,Motor function
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