Nusinersen improved respiratory function in spinal muscular atrophy type 2

2021 ◽  
Author(s):  
Ryuta Tanaka ◽  
Fujiko Fukushima ◽  
Keiichi Motoyama ◽  
Chie Kobayashi ◽  
Isho Izumi
Keyword(s):  
Spinal Muscular Atrophy ◽  
Respiratory Function ◽  
Muscular Atrophy ◽  
Spinal Muscular Atrophy Type

scholarly journals Improvement in Fine Manual Dexterity in Children with Spinal Muscular Atrophy Type 2 after Nusinersen Injection: A Case Series

Children ◽  
2021 ◽  
Vol 8 (11) ◽  
pp. 1039
Author(s):  
Minsu Gu ◽  
Hyun-Ho Kong
Keyword(s):  
Spinal Muscular Atrophy ◽  
Motor Function ◽  
Daily Living ◽  
Muscular Atrophy ◽  
Case Series ◽  
Manual Dexterity ◽  
Gross Motor ◽  
Spinal Muscular Atrophy Type ◽  
Gross Motor Function

Although nusinersen has been demonstrated to improve motor function in patients with spinal muscular atrophy (SMA), no studies have investigated its effect on fine manual dexterity. The present study aimed to investigate the ability of nusinersen to improve fine manual dexterity in patients with SMA type 2. A total of five patients with SMA type 2 were included. The Hammersmith Functional Motor Scale (expanded version) (HFMSE) and Purdue Pegboard (PP) tests were used to evaluate gross motor function and fine manual dexterity, respectively, until 18 months after nusinersen administration. HFMSE scores improved by 3–10 points (+13–53%) in all patients following nusinersen administration. PP scores also improved in all patients, from 4 to 9 points (+80–225%) in the preferred hand and from 3 to 7 points (+60–500%) in the non-preferred hand. These results suggest that nusinersen treatment improved both gross motor function and fine manual dexterity in children with SMA type 2. Addition of the PP test may aid in evaluating the fine manual dexterity essential for activities of daily living in these patients.

P.226Longitudinal study of the natural history of spinal muscular atrophy type 2 and 3

2019 ◽  
Vol 29 ◽  
pp. S132
Author(s):  
J. Exposito ◽  
D. Natera-de Benito ◽  
L. Carrera ◽  
A. Frongia ◽  
M. Alarcón ◽  
...  
Keyword(s):  
Natural History ◽  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Spinal Muscular Atrophy Type ◽  
History Of
Sign in / Sign up

Export Citation Format

Share Document