NEUROSAE 2018 ANNUAL MEETING EDITION (VOLUME 10, ISSUE 3)
QUESTION 10 OF 100
A 33-year-old woman with adult-onset spinal muscular dystrophy presents requesting enrollment in a clinical trial for nusinersen, a new FDA-approved medication for treatment of her condition. Which of the following is the appropriate route of administration?
E. Intrathecal **
** = Your answer
Nusinersen is an antisense oligonucleotide (ASO) that alters splicing of SMN2 mRNA and increases the amount of functional SMN protein produced, thus compensating for the genetic defect in the SMN1 gene. Nusinersen is administered intrathecally in four loading doses, every 14 days for the first three doses and the fourth dose administered 30 days after the third dose. A maintenance dose is administered every 4 months.
* Haché M, Swoboda KJ, Sethna N, et al. Intrathecal injections in children with spinal muscular atrophy: Nusinersen clinical trial experience. J Child Neurol. 2016 Jun;31(7):899-906.