Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy
Nature Neuroscience 20, 497 (2017).
doi:10.1038/nn.4508
Author: David R Corey
Nusinersen (Spinraza) is a recently approved drug for treating spinal muscular atrophy. Approval of nusinersen may signal new opportunities for using antisense oligonucleotides as treatments for devastating neurological diseases.
Source: Nature Neuroscience - Category: Neuroscience Authors: David R Corey Tags: News and Views Source Type: research