Nusinersen does not improve lung function in a cohort of children with spinal muscular atrophy–A single-center retrospective study
L Heitschmidt, L Pichlmaier, M Eckerland… - European Journal of …, 2021 - Elsevier
Objective To examine the effect of intrathecal application of nusinersen on the respiratory
function in terms of vital capacity in pediatric patients with spinal muscular atrophy (SMA).
SMA is characterized by on-going muscular atrophy and weakness that lead to respiratory
insufficiency. In recent years therapy with nusinersen has been shown to improve motor
function in patients with SMA. Methods We retrospectively analyzed data from 12 pediatric
patients (aged 4–12 years) with SMA II or III (7 walkers, 5 sitters) treated with nusinersen. We …
function in terms of vital capacity in pediatric patients with spinal muscular atrophy (SMA).
SMA is characterized by on-going muscular atrophy and weakness that lead to respiratory
insufficiency. In recent years therapy with nusinersen has been shown to improve motor
function in patients with SMA. Methods We retrospectively analyzed data from 12 pediatric
patients (aged 4–12 years) with SMA II or III (7 walkers, 5 sitters) treated with nusinersen. We …