25 research outputs found
Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy Therapy
Get PDFThe full text of this article can be found here: https://link.springer.com/article/10.1007/s40265-018-0868-
Reliability and Validity of Outcome Measures of In-Hospital and At-Home Visits in a Randomized, Double-Blind, Placebo-Controlled Trial for Spinal Muscular Atrophy
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Outcomes in patients with spinal muscular atrophy given nusinersen, onasemnogene abeparvovec or no treatment: an analysis based on restricted mean survival time
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Parentsâ perspectives on nusinersen treatment for children with spinal muscular atrophy
No full textNusinersen treatment significantly improves hand grip strength, hand motor function and MRC sum scores in adult patients with spinal muscular atrophy types 3 and 4
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