Antisense oligonucleotide shows promise in sma


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Access through your institution Buy or subscribe The antisense oligonucleotide nusinersen is safe and tolerable, and might improve motor function in children with spinal muscular atrophy


(SMA), according to a new phase I trial. SMA is caused by mutations in the _SMN1_ gene, and nusinersen compensates for loss of _SMN1_ function by altering the splicing of the mRNA encoded by


the paralogue _SMN2_. Trial participants who received a single 9 mg intrathecal injection of nusinersen showed significant increases in motor scores both 3 months and 9–14 months after


treatment, and the results indicate that the drug warrants further investigation in patients with SMA. This is a preview of subscription content, access via your institution ACCESS OPTIONS


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institutional subscriptions * Read our FAQs * Contact customer support REFERENCES * Chiriboga, C. A. _ et al_. Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal


muscular atrophy. _Neurology_ http://dx.doi.org/10.1212/WNL.0000000000002445 Download references Authors * Heather Wood View author publications You can also search for this author inPubMed


 Google Scholar RIGHTS AND PERMISSIONS Reprints and permissions ABOUT THIS ARTICLE CITE THIS ARTICLE Wood, H. Antisense oligonucleotide shows promise in SMA. _Nat Rev Neurol_ 12, 126 (2016).


https://doi.org/10.1038/nrneurol.2016.20 Download citation * Published: 26 February 2016 * Issue Date: March 2016 * DOI: https://doi.org/10.1038/nrneurol.2016.20 SHARE THIS ARTICLE Anyone


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