Type I SMA “new natural history”: long‐term data in nusinersen‐treated patients

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 (mea...

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發表在:Ann Clin Transl Neurol
Main Authors: Pane, Marika, Coratti, Giorgia, Sansone, Valeria A., Messina, Sonia, Catteruccia, Michela, Bruno, Claudio, Sframeli, Maria, Albamonte, Emilio, Pedemonte, Marina, D’Amico, Adele, Bravetti, Chiara, Berti, Beatrice, Palermo, Concetta, Leone, Daniela, Brigati, Giorgia, Tacchetti, Paola, Salmin, Francesca, De Sanctis, Roberto, Lucibello, Simona, Pera, Maria Carmela, Piastra, Marco, Genovese, Orazio, Bertini, Enrico, Vita, Gianluca, Tiziano, Francesco Danilo, Mercuri, Eugenio
格式: Artigo
語言:Inglês
出版: John Wiley and Sons Inc. 2021
主題:
Research Articles
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC7951096/
https://ncbi.nlm.nih.gov/pubmed/33547876
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/acn3.51276
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