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Expanded access program with Nusinersen in SMA type I in Italy: Strengths and pitfalls of a successful experience

2017; Elsevier BV; Volume: 27; Issue: 12 Linguagem: Inglês

10.1016/j.nmd.2017.09.006

1873-2364

Sonia Messina, Marika Pane, Valeria Sansone, Claudio Bruno, Michela Catteruccia, Giuseppe Vita, Concetta Palermo, Emilio Albamonte, Marina Pedemonte, Enrico Bertini, Luca Binetti, Eugenio Mercuri, Daniela Lauro, Anita Pallara, Simona Spinoglio, Maria Solinas, Grazia Zappa, Francesca Penno, Cristina Ponzanelli, Jacopo Casiraghi, Marcello Villanova, Danilo Tiziano, Anna Mandelli, Chiara Mastella, Alberto Mario Fontana, Anna Ambrosini, Daniela Leone, Roberto De Sanctis, Nicola Forcina, Marco Piastra, Orazio Genovese, Alessandro Pedicelli, Maria Sframeli, A Versaci, Imma Rulli, Eloisa Gitto, Cristina Faraone, Stefania La Foresta, Maria Macrì, Adele D’Amico, Giulia Colia, Anna Maria Bonetti, Adelina Carlesi, Renato Cutrera, Maria Beatrice Chiarini Testa, Marta Ferretti, Alberto Garaventa, Giovanni Montobbio, Carlo Gandolfo, Valentina Iurilli, Paola Tacchetti, Emilia Bobeica, Alessia D’Agostino, Alice Pirola, Sara Luppone, Elisa De Mattia, Elisa Falcier, Fabrizio Rao, Fausto Fedeli, Luca Mancini, Paolo Stoia, Maurizio Heinen, Valeria Cozzi, Beatrice Travaglia, Emma Mizzotti,

RNA modifications and cancer

We would like to share our experience with the expanded access program (EAP) using Nusinersen in infants and children with type 1 Spinal muscular atrophy (SMA). EAP is the use of an investigational new drug outside of a clinical trial in patients for the diagnosis, monitoring, or treatment of a serious disease or condition. EAP provides a pathway for patients to gain access to investigational drugs, biologics, and medical devices used to diagnose, monitor, or treat patients with serious diseases or conditions for which there are no comparable or satisfactory therapy options available outside of clinical trials. The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) facilitate the expanded access process.