Nusinersen for spinal muscular atrophy

Creative Commons Non Commercial CC BY-NC: This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 License (http://www.creativecommons.org/licenses/by-nc/4.0/) which permits non-commercial use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access pages (https://us.sagepub.com/en-us/nam/open-access-at-sage). Spinal muscular atrophy (SMA) therapy has been challenging for decades when considering the severity of the disorder and functional motor impairment on the one hand and the lack of pharmacological treatment options so far on the other. However, the recently approved antisense oligonucleotide (ASO) nusinersen now provides not only an upcoming and promising treatment option for SMA, but may also represent a general pharmacological approach and mechanism of action relevant for other neurodegenerative disorders.