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Expanded access program with Nusinersen in SMA type I in Italy: Strengths and pitfalls of a successful experience.
Neuromuscul Disord. 2017 Dec;27(12):1084-1086. doi: 10.1016/j.nmd.2017.09.006. Epub 2017 Sep 21.
Neuromuscul Disord. 2017.
PMID: 29132728
No abstract available.
An observational study of functional abilities in infants, children, and adults with type 1 SMA.
Pane M, Palermo C, Messina S, Sansone VA, Bruno C, Catteruccia M, Sframeli M, Albamonte E, Pedemonte M, D'Amico A, Brigati G, de Sanctis R, Coratti G, Lucibello S, Bertini E, Vita G, Danilo Tiziano F, Mercuri E; Italian EAP Working Group.
Pane M, et al.
Neurology. 2018 Aug 21;91(8):e696-e703. doi: 10.1212/WNL.0000000000006050. Epub 2018 Jul 25.
Neurology. 2018.
PMID: 30045959
Free PMC article.
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Intrathecal nusinersen treatment for SMA in a dedicated neuromuscular clinic: an example of multidisciplinary and integrated care.
Sansone VA, Albamonte E, Salmin F, Casiraghi J, Pirola A, Bettinelli M, Rao F, Mancini L, Tovaglieri N, Fedeli F, Stoia P, Heinen M, Cozzi V, Carraro E, Lunetta C, Di Bari A, Mercuri E; Italian EAP working group.
Sansone VA, et al.
Neurol Sci. 2019 Feb;40(2):327-332. doi: 10.1007/s10072-018-3622-9. Epub 2018 Nov 15.
Neurol Sci. 2019.
PMID: 30430317
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