Nusinersen improves walking distance and reduces fatigue in later-onset spinal muscular atrophy.
6-minute walk test
fatigue
neuromuscular junction
nusinersen
spinal muscular atrophy
Journal
Muscle & nerve
ISSN: 1097-4598
Titre abrégé: Muscle Nerve
Pays: United States
ID NLM: 7803146
Informations de publication
Date de publication:
10 2019
10 2019
Historique:
received:
07
02
2019
revised:
08
07
2019
accepted:
09
07
2019
pubmed:
13
7
2019
medline:
15
1
2020
entrez:
13
7
2019
Statut:
ppublish
Résumé
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. 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. 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%). These results support previous studies demonstrating clinically meaningful effects of nusinersen on motor function in children and adolescents with later-onset SMA.
Identifiants
pubmed: 31298747
doi: 10.1002/mus.26633
pmc: PMC6771553
doi:
Substances chimiques
Oligonucleotides
0
nusinersen
5Z9SP3X666
Types de publication
Clinical Trial, Phase I
Clinical Trial, Phase II
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
409-414Subventions
Organisme : NICHD NIH HHS
ID : K01 HD084690
Pays : United States
Commentaires et corrections
Type : CommentIn
Informations de copyright
© 2019 The Authors. Muscle & Nerve published by Wiley Periodicals, Inc.
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