Respiratory characteristics in children with spinal muscular atrophy type 1 receiving nusinersen.
Lena XiaoJackie ChiangMaria Luisa Castro CodesalHanna Katherine KolskiPrabhjot K BediFatema Al AmraniHernan D GonorazkyReshma Aminnull nullPublished in: Pediatric pulmonology (2022)
This study showed abnormal polysomnography parameters and need for ventilation despite nusinersen suggesting ongoing need for regular monitoring with polysomnography. Understanding the respiratory disease trajectory of children undergoing treatment with nusinersen will inform decision-making regarding optimal timing of ventilatory support initiation.