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01-12-2019 | Spinal Muscular Atrophy | Review article

Current evidence for treatment with nusinersen for spinal muscular atrophy: a systematic review

Authors: Antoon Meylemans, Jan De Bleecker

Published in: Acta Neurologica Belgica | Issue 4/2019

Abstract

Recent discovery of nusinersen, an antisense oligonucleotide drug, has provided encouragement for improving treatment of spinal muscular atrophy. No therapeutic options currently exist for this autosomal recessive motor neuron disorder. Nusinersen is developed for intrathecal use and binds to a specific sequence within the survival motor neuron 2 pre-messenger RNA, modifying the splicing process to promote expression of full-length survival motor neuron protein. We performed a MEDLINE and CENTRAL search to investigate the current evidence for treatment with nusinersen in patients with spinal muscular atrophy. Four papers were withheld, including two phase-3 randomized controlled trials, one phase-2 open-label clinical trial and one phase-1 open-label clinical trial. Outcome measures concerned improvement in motor function and milestones, as well as event-free survival and survival. Results of these trials are hopeful with significant and clinically meaningful improvement due to treatment with intrathecal nusinersen in patients with early- and later-onset spinal muscular atrophy, although this does not restore age-appropriate function. Intrathecal nusinersen has acceptable safety and tolerability. Further trials regarding long-term effects and safety aspects as well as trials including broader spinal muscular atrophy and age categories are required and ongoing.

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Title: Current evidence for treatment with nusinersen for spinal muscular atrophy: a systematic review
Authors: Antoon Meylemans
Jan De Bleecker
Publication date: 01-12-2019
Publisher: Springer International Publishing
Keyword: Spinal Muscular Atrophy
Published in: Acta Neurologica Belgica / Issue 4/2019
Print ISSN: 0300-9009
Electronic ISSN: 2240-2993
DOI: https://doi.org/10.1007/s13760-019-01199-z

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Current evidence for treatment with nusinersen for spinal muscular atrophy: a systematic review

Abstract

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