دورية أكاديمية
Type I SMA 'new natural history': long‐term data in nusinersen‐treated patients
| العنوان: | Type I SMA 'new natural history': long‐term data in nusinersen‐treated patients |
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| المؤلفون: | Marika Pane, Giorgia Coratti, Valeria A. Sansone, Sonia Messina, Michela Catteruccia, Claudio Bruno, Maria Sframeli, Emilio Albamonte, Marina Pedemonte, Adele D’Amico, Chiara Bravetti, Beatrice Berti, Concetta Palermo, Daniela Leone, Giorgia Brigati, Paola Tacchetti, Francesca Salmin, Roberto De Sanctis, Simona Lucibello, Maria Carmela Pera, Marco Piastra, Orazio Genovese, Enrico Bertini, Gianluca Vita, Francesco Danilo Tiziano, Eugenio Mercuri, the Italian EAP Working Group |
| المصدر: | Annals of Clinical and Translational Neurology, Vol 8, Iss 3, Pp 548-557 (2021) |
| بيانات النشر: | Wiley, 2021. |
| سنة النشر: | 2021 |
| المجموعة: | LCC:Neurosciences. Biological psychiatry. Neuropsychiatry LCC:Neurology. Diseases of the nervous system |
| مصطلحات موضوعية: | Neurosciences. Biological psychiatry. Neuropsychiatry, RC321-571, Neurology. Diseases of the nervous system, RC346-429 |
| الوصف: | Abstract Objective The aim of this paper was to report the 2‐year follow‐up in type I patients treated with Nusinersen and to assess whether possible changes in motor function are related to the subtype, age, or SMN2 copy number. Methods Sixty‐eight patients, with ages ranging from 0.20 to 15.92 years (mean: 3.96; standard deviation: +3.90) were enrolled in the study. All patients were assessed using the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) and the developmental section of the Hammersmith Infant Neurological Examination (HINE‐2) at the time they started treatment and 12 and 24 months after that. Results For both CHOP and HINE‐2 repeated measures analysis of variance showed a significant difference (P |
| نوع الوثيقة: | article |
| وصف الملف: | electronic resource |
| اللغة: | English |
| تدمد: | 2328-9503 |
| العلاقة: | https://doaj.org/toc/2328-9503Test |
| DOI: | 10.1002/acn3.51276 |
| الوصول الحر: | https://doaj.org/article/21f37327eb0f4e088a3f71a02ca0497dTest |
| رقم الانضمام: | edsdoj.21f37327eb0f4e088a3f71a02ca0497d |
| قاعدة البيانات: | Directory of Open Access Journals |
Full Text Finder | تدمد: | 23289503 |
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| DOI: | 10.1002/acn3.51276 |