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

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

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 yea...

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Autori principali: 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
Natura: Articolo
Lingua:English
Pubblicazione: Wiley 2021-03-01
Serie:Annals of Clinical and Translational Neurology
Accesso online:https://doi.org/10.1002/acn3.51276

Accesso online

https://doi.org/10.1002/acn3.51276

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