Nusinersen efficacy data for 24-month in type 2 and 3 spinal muscular atrophy.
Journal
Annals of clinical and translational neurology
ISSN: 2328-9503
Titre abrégé: Ann Clin Transl Neurol
Pays: United States
ID NLM: 101623278
Informations de publication
Date de publication:
03 2022
03 2022
Historique:
revised:
14
01
2022
received:
30
12
2021
accepted:
14
01
2022
pubmed:
16
2
2022
medline:
4
5
2022
entrez:
15
2
2022
Statut:
ppublish
Résumé
The study reports real world data in type 2 and 3 SMA patients treated for at least 2 years with nusinersen. Increase in motor function was observed after 12 months and during the second year. The magnitude of change was variable across age and functional subgroup, with the largest changes observed in young patients with higher function at baseline. When compared to natural history data, the difference between study cohort and untreated patients swas significant on both Hammersmith Functional Motor Scale and Revised Upper Limb Module both at 12 months and at 24 months.
Identifiants
pubmed: 35166467
doi: 10.1002/acn3.51514
pmc: PMC8935309
doi:
Substances chimiques
Oligonucleotides
0
nusinersen
5Z9SP3X666
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
404-409Commentaires et corrections
Type : CommentIn
Informations de copyright
© 2022 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association.
Références
Nat Rev Neurol. 2020 Dec;16(12):706-715
pubmed: 33057172
Ann Clin Transl Neurol. 2021 Aug;8(8):1622-1634
pubmed: 34165911
J Neuromuscul Dis. 2018;5(2):159-166
pubmed: 29865090
Neuromuscul Disord. 2020 Sep;30(9):756-764
pubmed: 32900576
Orphanet J Rare Dis. 2021 Oct 13;16(1):430
pubmed: 34645478
Lancet Neurol. 2021 Apr;20(4):284-293
pubmed: 33743238
Neurology. 2019 May 21;92(21):e2492-e2506
pubmed: 31019106
Ann Clin Transl Neurol. 2021 Mar;8(3):548-557
pubmed: 33547876
Neuromuscul Disord. 2019 Oct;29(10):794-799
pubmed: 31558335
JAMA Neurol. 2021 Jul 1;78(7):834-841
pubmed: 33999158
Value Health. 2021 Jun;24(6):759-769
pubmed: 34119073
Neuromuscul Disord. 2021 Jul;31(7):596-602
pubmed: 34099377
Muscle Nerve. 2017 Jun;55(6):869-874
pubmed: 27701745
J Neuromuscul Dis. 2021;8(1):101-108
pubmed: 33074187
Muscle Nerve. 2019 Apr;59(4):426-430
pubmed: 30677148
Muscle Nerve. 2021 May;63(5):668-677
pubmed: 33501671
Neuromuscul Disord. 2021 Mar;31(3):183-193
pubmed: 33608138
J Neuromuscul Dis. 2021;8(2):209-216
pubmed: 33427694
Neuromuscul Disord. 2007 Oct;17(9-10):693-7
pubmed: 17658255
Neuromuscul Disord. 2020 Sep;30(9):765-771
pubmed: 32893082
Ann Neurol. 2020 Dec;88(6):1109-1117
pubmed: 32926458
Neurology. 2019 Sep 24;93(13):e1241-e1247
pubmed: 31451515