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Nusinersen efficacy data for 24‐month in type 2 and 3 spinal muscular atrophy
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...
| Autores principales: | , , , , , , , , , , , , , , , , , , , , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
John Wiley and Sons Inc.
2022
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8935309/ https://www.ncbi.nlm.nih.gov/pubmed/35166467 http://dx.doi.org/10.1002/acn3.51514 |
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| author | Pane, Marika Coratti, Giorgia Pera, Maria Carmela Sansone, Valeria A. Messina, Sonia d'Amico, Adele Bruno, Claudio Salmin, Francesca Albamonte, Emilio De Sanctis, Roberto Sframeli, Maria Di Bella, Vincenzo Morando, Simone Palermo, Concetta Frongia, Anna Lia Antonaci, Laura Capasso, Anna Catteruccia, Michela Longo, Antonella Ricci, Martina Cutrona, Costanza Pirola, Alice Bravetti, Chiara Pedemonte, Marina Brolatti, Noemi Bertini, Enrico Mercuri, Eugenio |
| author_facet | Pane, Marika Coratti, Giorgia Pera, Maria Carmela Sansone, Valeria A. Messina, Sonia d'Amico, Adele Bruno, Claudio Salmin, Francesca Albamonte, Emilio De Sanctis, Roberto Sframeli, Maria Di Bella, Vincenzo Morando, Simone Palermo, Concetta Frongia, Anna Lia Antonaci, Laura Capasso, Anna Catteruccia, Michela Longo, Antonella Ricci, Martina Cutrona, Costanza Pirola, Alice Bravetti, Chiara Pedemonte, Marina Brolatti, Noemi Bertini, Enrico Mercuri, Eugenio |
| author_sort | Pane, Marika |
| collection | PubMed |
| description | 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. |
| format | Online Article Text |
| id | pubmed-8935309 |
| institution | National Center for Biotechnology Information |
| language | English |
| publishDate | 2022 |
| publisher | John Wiley and Sons Inc. |
| record_format | MEDLINE/PubMed |
| spelling | pubmed-89353092022-03-24 Nusinersen efficacy data for 24‐month in type 2 and 3 spinal muscular atrophy Pane, Marika Coratti, Giorgia Pera, Maria Carmela Sansone, Valeria A. Messina, Sonia d'Amico, Adele Bruno, Claudio Salmin, Francesca Albamonte, Emilio De Sanctis, Roberto Sframeli, Maria Di Bella, Vincenzo Morando, Simone Palermo, Concetta Frongia, Anna Lia Antonaci, Laura Capasso, Anna Catteruccia, Michela Longo, Antonella Ricci, Martina Cutrona, Costanza Pirola, Alice Bravetti, Chiara Pedemonte, Marina Brolatti, Noemi Bertini, Enrico Mercuri, Eugenio Ann Clin Transl Neurol Brief Communication 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. John Wiley and Sons Inc. 2022-02-15 /pmc/articles/PMC8935309/ /pubmed/35166467 http://dx.doi.org/10.1002/acn3.51514 Text en © 2022 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ (https://creativecommons.org/licenses/by-nc-nd/4.0/) License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made. |
| spellingShingle | Brief Communication Pane, Marika Coratti, Giorgia Pera, Maria Carmela Sansone, Valeria A. Messina, Sonia d'Amico, Adele Bruno, Claudio Salmin, Francesca Albamonte, Emilio De Sanctis, Roberto Sframeli, Maria Di Bella, Vincenzo Morando, Simone Palermo, Concetta Frongia, Anna Lia Antonaci, Laura Capasso, Anna Catteruccia, Michela Longo, Antonella Ricci, Martina Cutrona, Costanza Pirola, Alice Bravetti, Chiara Pedemonte, Marina Brolatti, Noemi Bertini, Enrico Mercuri, Eugenio Nusinersen efficacy data for 24‐month in type 2 and 3 spinal muscular atrophy |
| title | Nusinersen efficacy data for 24‐month in type 2 and 3 spinal muscular atrophy |
| title_full | Nusinersen efficacy data for 24‐month in type 2 and 3 spinal muscular atrophy |
| title_fullStr | Nusinersen efficacy data for 24‐month in type 2 and 3 spinal muscular atrophy |
| title_full_unstemmed | Nusinersen efficacy data for 24‐month in type 2 and 3 spinal muscular atrophy |
| title_short | Nusinersen efficacy data for 24‐month in type 2 and 3 spinal muscular atrophy |
| title_sort | nusinersen efficacy data for 24‐month in type 2 and 3 spinal muscular atrophy |
| topic | Brief Communication |
| url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8935309/ https://www.ncbi.nlm.nih.gov/pubmed/35166467 http://dx.doi.org/10.1002/acn3.51514 |
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