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This study aims to assess how lung function and respiratory muscle strength change during treatment with nusinersen in spinal muscular atrophy patients.

May 9, 2026Neurology1 citationsOpen Access

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Lung Function and Respiratory Muscle Strength in Symptomatic Children and Adults With Spinal Muscular Atrophy Treated With Nusinersen

FVFemke VercoelenUniversity Medical Center Utrecht LALieke A.P. ArtsUniversity Medical Center Utrecht MBMarit E.M. de BruijneUniversity Medical Center Utrecht

Key Points

This study aims to assess how lung function and respiratory muscle strength change during treatment with nusinersen in spinal muscular atrophy patients.
Evaluated longitudinal changes in lung function and respiratory muscle strength
Focused on patients with symptomatic spinal muscular atrophy
Utilized nusinersen treatment as a primary intervention
Improvements in lung function were observed in participants receiving nusinersen therapy
Respiratory muscle strength enhanced following treatment
The study highlights the potential benefits of nusinersen beyond motor function.

Abstract

Spinal muscular atrophy (SMA) causes progressive muscle weakness. Respiratory muscle weakness results in progressive lung function decline, scoliosis, and chest wall deformities that predispose to respiratory failure. The efficacy of survival motor neuron protein–augmenting therapies on motor function in patients with SMA has been reported extensively, but how respiratory function changes under treatment remains underexplored. The aim of this study was to evaluate longitudinal changes in lung function and respiratory muscle strength during nusinersen treatment.

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Lung Function and Respiratory Muscle Strength in Symptomatic Children and Adults With Spinal Muscular Atrophy Treated With Nusinersen

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