Effect of nusinersen on respiratory function in paediatric spinal muscular atrophy types 1-3.
Archana ChackoPeter D SlyRobert S WareNelufa BegumSean DeeganNicole ThomasLeanne M GauldPublished in: Thorax (2021)
The first year of nusinersen treatment saw reduced lung function decline (especially in type 2) and improvement in AHI.