Nusinersen (ISIS-SMNRx or ISIS 396443) is an antisense oligonucleotide drug 
administered intrathecally to treat spinal muscular atrophy. We summarize lumbar 
puncture experience in children with spinal muscular atrophy during a phase 1 
open-label study of nusinersen and its extension. During the studies, 73 lumbar 
punctures were performed in 28 patients 2 to 14 years of age with type 2/3 
spinal muscular atrophy. No complications occurred in 50 (68%) lumbar punctures; 
in 23 (32%) procedures, adverse events were attributed to lumbar puncture. Most 
common adverse events were headache (n = 9), back pain (n = 9), and post-lumbar 
puncture syndrome (n = 8). In a subgroup analysis, adverse events were more 
frequent in older children, children with type 3 spinal muscular atrophy, and 
with a 21- or 22-gauge needle compared to a 24-gauge needle or smaller. Lumbar 
punctures were successfully performed in children with spinal muscular atrophy; 
lumbar puncture-related adverse event frequency was similar to that previously 
reported in children.