Journal
PEDIATRIC PULMONOLOGY
Volume 56, Issue 1, Pages 299-306Publisher
WILEY
DOI: 10.1002/ppul.25142
Keywords
lung function; motor function; nusinersen; respiratory muscles; spinal muscular atrophy
Categories
Ask authors/readers for more resources
The study found that respiratory muscle performance in children with SMA Type 2 significantly improved after six injections of nusinersen, and there was also a significant improvement in motor function tests.
Introduction Nusinersen is associated with an improvement in motor function in children with spinal muscular atrophy (SMA) but data on respiratory muscles strength are scarce. Respiratory muscles performance and lung function were evaluated in children with SMA 1c and 2 after six injections of nusinersen (M14). Results from patients with SMA2 were compared with data of age-matched historical controls. Motor function tests (MFM and HINE-2) were assessed at baseline and M14 in the treated patients. Results Sixteen children (2 SMA Type 1c and 14 SMA Type 2), mean age 9.4 +/- 2.3 years, were included. The data of 14 historical SMA 2 controls (mean age 9.3 +/- 1.9 years) were gathered. The strength of the global inspiratory muscles of SMA 2 treated with nusinersen, assessed on maximal static inspiratory pressure, forced vital capacity, and esophageal pressure during a maximal sniff was significantly better compared with historical controls (p < .05). A significant improvement in MFM and HINE-2 was observed in the patients with 16 SMA treated with nusinersen after 14 months as compared with baseline. Conclusion In children with SMA Type 2, respiratory muscle performance was significantly better after six injections of nusinersen as compared with age-matched SMA Type 2 historical controls.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available