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Safety, tolerability, and efficacy of a widely available nusinersen program for Polish children with Spinal Muscular Atrophy

Katarzyna Kotulska,Dariusz Chmielewski,Maria Mazurkiewicz-Bełdzińska,Katarzyna Tomaszek,Katarzyna Pierzchlewicz,Daniel Rabczenko,Łukasz Przysło,Agnieszka Biedroń,Elżbieta Czyżyk,Barbara Steinborn,Jerzy Pietruszewski,Leszek Boćkowski, Dorota Cichosz,Magdalena Dudzińska, Elżbieta Gadowska, Elżbieta Młynarczyk, Mirosław Jasiński, Anna Masztalerz, Agnieszka Kempisty,Anna Kostera-Pruszczyk

European Journal of Paediatric Neurology（2022）

Cited 2|Views10
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Abstract
Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder with limited treatment options. Nusinersen is the first disease-modifying therapy to treat children and adults with SMA. This study aimed to review the safety, tolerability, and efficacy data of a nusinersen treatment program in Polish children.
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Key words
Spinal muscular atrophy,SMA,Nusinersen
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