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

Ando, S; Suzuki, S; Okubo, S; Ohuchi, K; Takahashi, K; Nakamura, S; Shimazawa, M; Fuji, K; Hara, H

Hara, H (corresponding author), Gifu Pharmaceut Univ, Dept Biofunct Evaluat, Mol Pharmacol, 1-25-4 Daigaku Nishi, Gifu 5011196, Japan.

SCIENTIFIC REPORTS, 2020; 10 (1):

Abstract

Spinal muscular atrophy (SMA) is a motor neuron disease, typically resulting from loss-of-function mutations in the survival motor neuron 1 (SMN1) gen......

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