History of development of the life-saving drug “Nusinersen” in spinal muscular atrophy

History of development of the life-saving drug “Nusinersen” in spinal muscular atrophy

Spinal muscular atrophy (SMA) is an autosomal recessive disorder with an incidence of 1/6,000–1/10,000 and is the leading fatal disease among infants. Previously, there was no effective treatment for SMA. The first effective drug, nusinersen, was approved by the US FDA in December 2016, providing ho...

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Bibliographic Details
Main Authors: Jiaying Qiu, Liucheng Wu, Ruobing Qu, Tao Jiang, Jialin Bai, Lei Sheng, Pengchao Feng, Junjie Sun
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-08-01
Series:Frontiers in Cellular Neuroscience
Subjects:
spinal muscular atrophy
SMN1/2
antisense oligonucleotide
splicing regulation
nusinersen
Online Access:https://www.frontiersin.org/articles/10.3389/fncel.2022.942976/full

Internet

https://www.frontiersin.org/articles/10.3389/fncel.2022.942976/full

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