Study on the efficacy, safety, and biomarkers of nusinersen in type II and III spinal muscular atrophy in children

Study on the efficacy, safety, and biomarkers of nusinersen in type II and III spinal muscular atrophy in children

Introduction/aimsThe time span for the approval of nusinersen to treat SMA remains short. Most studies on the efficacy and safety of this drug within clinical trials, are lacking real-world research data. This study is based on real-world studies of SMA patients in children with type II and III SMA...

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Bibliographic Details
Main Authors: Liyuan Chen, Fen Liu, Danna Fang, Jianwei Li
Format: Article
Language:English
Published: Frontiers Media S.A. 2023-12-01
Series:Frontiers in Pediatrics
Subjects:
nusinersen
SMA
motor function
platelet
urinary protein
Online Access:https://www.frontiersin.org/articles/10.3389/fped.2023.1294405/full

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