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

Marika Pane, Eugenio Maria Mercuri, Francesco Danilo Tiziano, Marco Piastra, Michela Catteruccia, Concetta Palermo, Emilio Albamonte, Enrico Silvio Bertini, Jacopo Luca Casiraghi, Alberto Fontana, Daniela Leone, Roberto De Sanctis, Orazio Genovese, Alessandro Pedicelli, Adele D'Amico, Sonia Messina, Valeria Sansone, Claudio Bruno, Giuseppe Vita, Marina PedemonteEnrico Bertini, Luca Binetti, Eugenio Mercuri

Research output: Contribution to journalArticle

27 Citations (Scopus)

Abstract

Expanded access program with Nusinersen in SMA type I
Original languageEnglish
Pages (from-to)1084-1086
Number of pages3
JournalNeuromuscular Disorders
Volume27
DOIs
Publication statusPublished - 2017

Keywords

  • Genetics (clinical)
  • Neurology
  • Neurology (clinical)
  • Pediatrics, Perinatology and Child Health

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