Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

Corey, DR

Corey, DR (reprint author), UT Southwestern, Dept Pharmacol, Dallas, TX 75235 USA.; Corey, DR (reprint author), UT Southwestern, Dept Biochem, Dallas, TX 75235 USA.

NATURE NEUROSCIENCE, 2017; 20 (4): 497

Abstract

Nusinersen (Spinraza) is a recently approved drug for treating spinal muscular atrophy. Approval of nusinersen may signal new opportunities for using ......

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