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...

Полное описание

Библиографические подробности
Главные авторы: Jiaying Qiu, Liucheng Wu, Ruobing Qu, Tao Jiang, Jialin Bai, Lei Sheng, Pengchao Feng, Junjie Sun
Формат: Статья
Язык:English
Опубликовано: Frontiers Media S.A. 2022-08-01
Серии:Frontiers in Cellular Neuroscience
Предметы:
spinal muscular atrophy
SMN1/2
antisense oligonucleotide
splicing regulation
nusinersen
Online-ссылка: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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