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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...

Ausführliche Beschreibung

Gespeichert in:
Hauptverfasser: Jiaying Qiu, Liucheng Wu, Ruobing Qu, Tao Jiang, Jialin Bai, Lei Sheng, Pengchao Feng, Junjie Sun
Format: Artigo
Sprache:Inglês
Veröffentlicht: Frontiers Media S.A. 2022-08-01
Schriftenreihe:Frontiers in Cellular Neuroscience
Schlagworte:
spinal muscular atrophy
SMN1/2
antisense oligonucleotide
splicing regulation
nusinersen
Online-Zugang:https://www.frontiersin.org/articles/10.3389/fncel.2022.942976/full
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