Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy
Sign Up to like & getrecommendations! Published in 2017 at "Nature Neuroscience"
DOI: 10.1038/nn.4508
Abstract: 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. read more here.
Keywords: spinal muscular; drug; antisense oligonucleotide; nusinersen antisense ... See more keywords