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Article

Discovery of a CNS penetrant small molecule SMN2 splicing modulator with improved tolerability for spinal muscular atrophy.

Scientific reports - 15 Oct 2020

Ando Shiori, Suzuki Shunya, Okubo Shoichi, Ohuchi Kazuki, Takahashi Kei, Nakamura Shinsuke, Shimazawa Masamitsu, Fuji Koji, Hara Hideaki

Abstract excerpt

Spinal muscular atrophy (SMA) is a motor neuron disease, typically resulting from loss-of-function mutations in the survival motor neuron 1 (SMN1) gene. Nusinersen/SPINRAZA, a splice-switching oligonucleotide that modulates SMN2 (a paralog of SMN1) splicing and consequently increases SMN protein levels, has a therapeutic effect for SMA. Previously reported small-molecule SMN2 splicing modulators such as...

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Identifiers

PMID: 33060681
DOI: 10.1038/s41598-020-74346-9
PMCID: PMC7562719

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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