1. Expanded access program with Nusinersen in SMA type I in Italy: Strengths and pitfalls of a successful experience
- Author
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Emilia Bobeica, Cristina Ponzanelli, Paolo Stoia, Emilio Albamonte, Valeria A. Sansone, Fabrizio Rao, Claudio Bruno, Cristina Faraone, Danilo Tiziano, Valeria Cozzi, Marika Pane, Elisa De Mattia, Anna Maria Bonetti, Maria Sframeli, Marcello Villanova, Orazio Genovese, Anna Mandelli, Chiara Mastella, Marco Piastra, Sara Luppone, Francesca Penno, Adele D'Amico, Sonia Messina, Anna Ambrosini, Alessandro Pedicelli, Antonio Versaci, Stefania La Foresta, Luca Binetti, Alessia D'Agostino, Nicola Forcina, Giulia Colia, Elisa Falcier, Maria Macrì, Maurizio Heinen, Luca Mancini, Jacopo Casiraghi, Maria Beatrice Chiarini, Carlo Gandolfo, Concetta Palermo, Marta Ferretti, Maria Letizia Solinas, Giuseppe Vita, Imma Rulli, Giovanni Montobbio, Anita Pallara, Alice Pirola, Daniela Leone, Michela Catteruccia, Grazia Zappa, Renato Cutrera, Paola Tacchetti, Adelina Carlesi, Eloisa Gitto, Daniela Lauro, Alberto Fontana, Valentina Iurilli, Emma Mizzotti, Beatrice Travaglia, Enrico Bertini, Eugenio Mercuri, Fausto Fedeli, Marina Pedemonte, Roberto De Sanctis, Alberto Garaventa, and Simona Spinoglio
- Subjects
0301 basic medicine ,MEDLINE ,Oligonucleotides ,Spinal Muscular Atrophies of Childhood ,Pediatrics ,03 medical and health sciences ,0302 clinical medicine ,Settore MED/41 - ANESTESIOLOGIA ,Medicine ,Humans ,Genetics (clinical) ,Medical education ,business.industry ,Pediatrics, Perinatology and Child Health ,Neurology ,Neurology (clinical) ,Perinatology and Child Health ,SMA ,Survival of Motor Neuron 1 Protein ,030104 developmental biology ,Italy ,Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA ,Expanded access ,Nusinersen ,Expanded access program ,business ,030217 neurology & neurosurgery - Published
- 2017