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Evaluates lung function changes in spinal muscular atrophy patients undergoing nusinersen treatment, indicating treatment effects on respiratory health.
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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Vercoelen et al. (2026) studied this question.
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