A comprehensive institutional overview of intrathecal nusinersen injections for spinal muscular atrophy
- 18 July 2018
- journal article
- research article
- Published by Springer Science and Business Media LLC in Pediatric Radiology
- Vol. 48 (12), 1797-1805
- https://doi.org/10.1007/s00247-018-4206-9
Abstract
Spinal muscular atrophy (SMA) is an autosomal-recessive neuromuscular disorder resulting in progressive muscle weakness. In December 2016, the U.S. Food and Drug Administration approved the first treatment for SMA, a drug named nusinersen (Spinraza) that is administered intrathecally. However many children with SMA have neuromuscular scoliosis or spinal instrumentation resulting in challenging intrathecal access. Therefore alternative routes must be considered in these complex patients.Keywords
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