Evaluation of Children with SMA Type 1 Under Treatment with Nusinersen within the Expanded Access Program in Germany.

Astrid Pechmann,Thorsten Langer,David Schorling,Sabine Stein,Sibylle Vogt,Ulrike Schara,Heike Kölbel,Oliver Schwartz,Andreas Hahn, Kerstin Giese,Jessika Johannsen,Jonas Denecke,Claudia Weiß,Manuela Theophil,Janbernd Kirschner

Journal of neuromuscular diseases（2018）

引用 100|浏览20

暂无评分

摘要

When analyzing a broad spectrum of SMA type 1 patients, many children showed an improvement of motor function after six months of treatment with nusinersen, which is generally not expected within the natural course of the disease. Long-term observation and follow-up of patients with later onset types of SMA are crucial to understand the clinical impact of treatment with nusinersen.

查看译文

关键词

SMArtCARE,Spinal muscular atrophy,motor function,motor milestones,nusinersen

AI 理解论文

溯源树

样例

生成溯源树，研究论文发展脉络

Chat Paper

正在生成论文摘要