Back to Search
Start Over
Nusinersen improves walking distance and reduces fatigue in later‐onset spinal muscular atrophy
- Source :
- Muscle & Nerve
- Publication Year :
- 2019
- Publisher :
- Wiley, 2019.
-
Abstract
- Introduction Ambulatory individuals with spinal muscular atrophy (SMA) experience muscle weakness, gait impairments, and fatigue that affect their walking ability. Improvements have been observed in motor function in children treated with nusinersen, but its impact on fatigue has not been studied. Methods Post hoc analyses were used to examine changes in 6‐minute walk test (6MWT) distance and fatigue in children and adolescents with SMA type II and III who received their first dose of nusinersen in the phase Ib/IIa, open‐label CS2 study and were ambulatory during CS2 or the extension study, CS12. Results Fourteen children performed the 6MWT. Median (25th, 75th percentile) distance walked increased over time by 98.0 (62.0, 135.0) meters at day 1050, whereas median fatigue changed by −3.8% (−19.7%, 1.4%). Discussion These results support previous studies demonstrating clinically meaningful effects of nusinersen on motor function in children and adolescents with later‐onset SMA.
- Subjects :
- Male
0301 basic medicine
6‐minute walk test
medicine.medical_specialty
Percentile
Adolescent
Physiology
Oligonucleotides
Walk Test
Walking
030105 genetics & heredity
Muscular Atrophy, Spinal
03 medical and health sciences
Cellular and Molecular Neuroscience
0302 clinical medicine
Physical medicine and rehabilitation
Physiology (medical)
medicine
Humans
Child
spinal muscular atrophy
Aged
neuromuscular junction
business.industry
nusinersen
Infant
Muscle weakness
Spinal muscular atrophy
medicine.disease
SMA
Gait
Walk test
Child, Preschool
Clinical Research Short Report
Ambulatory
Clinical Research Short Reports
Female
fatigue
Nusinersen
Neurology (clinical)
medicine.symptom
business
030217 neurology & neurosurgery
Subjects
Details
- ISSN :
- 10974598 and 0148639X
- Volume :
- 60
- Database :
- OpenAIRE
- Journal :
- Muscle & Nerve
- Accession number :
- edsair.doi.dedup.....ac1d596a49ceffccfbb8d0b1cf721eb4