Nusinersen efficacy data for 24-month in type 2 and 3 spinal muscular atrophy
© 2022 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association..
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.
| Errataetall: |
CommentIn: Nat Rev Neurol. 2022 Apr;18(4):187. - PMID 35228704 |
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| Medienart: |
E-Artikel |
| Erscheinungsjahr: |
2022 |
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| Erschienen: |
2022 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:9 |
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| Enthalten in: |
Annals of clinical and translational neurology - 9(2022), 3 vom: 15. März, Seite 404-409 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Pane, Marika [VerfasserIn] |
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| Links: |
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| Themen: |
5Z9SP3X666 |
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| Anmerkungen: |
Date Completed 03.05.2022 Date Revised 03.05.2022 published: Print-Electronic CommentIn: Nat Rev Neurol. 2022 Apr;18(4):187. - PMID 35228704 Citation Status MEDLINE |
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| doi: |
10.1002/acn3.51514 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM336985320 |
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| 245 | 1 | 0 | |a Nusinersen efficacy data for 24-month in type 2 and 3 spinal muscular atrophy |
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| 500 | |a CommentIn: Nat Rev Neurol. 2022 Apr;18(4):187. - PMID 35228704 | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a © 2022 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association. | ||
| 520 | |a 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 | ||
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