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: Tags: News and Views Source Type: research