Photo from wikipedia
Spinal muscular atrophy type 1 (SMA1) is a neuromuscular disorder with a natural history of chronic respiratory failure and death during infancy without ventilation. Recently, disease‐modifying therapies such as nusinersen… Click to show full abstract
Spinal muscular atrophy type 1 (SMA1) is a neuromuscular disorder with a natural history of chronic respiratory failure and death during infancy without ventilation. Recently, disease‐modifying therapies such as nusinersen have improved disease trajectory. However, objective data on the trajectory of polysomnography outcomes, the relationship between motor scores and respiratory parameters, respiratory technology dependence and healthcare utilization in children with SMA1 remain to be elucidated.
Journal Title: Pediatric Pulmonology
Year Published: 2022
Link to full text (if available)
Share on Social Media: Sign Up to like & get
recommendations!