Current evidence for treatment with nusinersen for spinal muscular atrophy: a systematic review
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...
Paper Details
Title
Current evidence for treatment with nusinersen for spinal muscular atrophy: a systematic review
Published Date
Aug 6, 2019
Journal
Volume
119
Issue
4
Pages
523 - 533
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