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

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 Letizia Solinas, Grazia Zappa, Francesca Penno, Cristina Ponzanelli, Jacopo CasiraghiMarcello Villanova, Danilo Tiziano, Anna Mandelli, Chiara Mastella, Alberto Fontana, Anna Ambrosini, Daniela Leone, Roberto de Sanctis, Nicola Forcina, Marco Piastra, Orazio Genovese, Alessandro Pedicelli, Maria Sframeli, Antonio 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, Marta Ferretti, Alberto Garaventa, Giovanni Montobbio, Carlo Gandolfo, Valentina Iurilli, on behalf of the Italian EAP working Group

Research output: Contribution to journalEditorial

Original languageEnglish
Pages (from-to)1084-1086
Number of pages3
JournalNeuromuscular Disorders
Volume27
Issue number12
DOIs
Publication statusPublished - Dec 1 2017

ASJC Scopus subject areas

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

Cite this

Messina, S., Pane, M., Sansone, V., Bruno, C., Catteruccia, M., Vita, G., ... on behalf of the Italian EAP working Group (2017). Expanded access program with Nusinersen in SMA type I in Italy: Strengths and pitfalls of a successful experience. Neuromuscular Disorders, 27(12), 1084-1086. https://doi.org/10.1016/j.nmd.2017.09.006

Expanded access program with Nusinersen in SMA type I in Italy : Strengths and pitfalls of a successful experience. / Messina, Sonia; Pane, Marika; Sansone, Valeria; Bruno, Claudio; Catteruccia, Michela; Vita, Giuseppe; Palermo, Concetta; Albamonte, Emilio; Pedemonte, Marina; Bertini, Enrico; Binetti, Luca; Mercuri, Eugenio; Lauro, Daniela; Pallara, Anita; Spinoglio, Simona; Solinas, Maria Letizia; Zappa, Grazia; Penno, Francesca; Ponzanelli, Cristina; Casiraghi, Jacopo; Villanova, Marcello; Tiziano, Danilo; Mandelli, Anna; Mastella, Chiara; Fontana, Alberto; Ambrosini, Anna; Leone, Daniela; de Sanctis, Roberto; Forcina, Nicola; Piastra, Marco; Genovese, Orazio; Pedicelli, Alessandro; Sframeli, Maria; Versaci, Antonio; Rulli, Imma; Gitto, Eloisa; Faraone, Cristina; La Foresta, Stefania; Macrì, Maria; D'Amico, Adele; Colia, Giulia; Bonetti, Anna Maria; Carlesi, Adelina; Cutrera, Renato; Chiarini, Maria Beatrice; Ferretti, Marta; Garaventa, Alberto; Montobbio, Giovanni; Gandolfo, Carlo; Iurilli, Valentina; on behalf of the Italian EAP working Group.

In: Neuromuscular Disorders, Vol. 27, No. 12, 01.12.2017, p. 1084-1086.

Research output: Contribution to journalEditorial

Messina, S, Pane, M, Sansone, V, Bruno, C, Catteruccia, M, Vita, G, Palermo, C, Albamonte, E, Pedemonte, M, Bertini, E, Binetti, L, Mercuri, E, Lauro, D, Pallara, A, Spinoglio, S, Solinas, ML, Zappa, G, Penno, F, Ponzanelli, C, Casiraghi, J, Villanova, M, Tiziano, D, Mandelli, A, Mastella, C, Fontana, A, Ambrosini, A, Leone, D, de Sanctis, R, Forcina, N, Piastra, M, Genovese, O, Pedicelli, A, Sframeli, M, Versaci, A, Rulli, I, Gitto, E, Faraone, C, La Foresta, S, Macrì, M, D'Amico, A, Colia, G, Bonetti, AM, Carlesi, A, Cutrera, R, Chiarini, MB, Ferretti, M, Garaventa, A, Montobbio, G, Gandolfo, C, Iurilli, V & on behalf of the Italian EAP working Group 2017, 'Expanded access program with Nusinersen in SMA type I in Italy: Strengths and pitfalls of a successful experience', Neuromuscular Disorders, vol. 27, no. 12, pp. 1084-1086. https://doi.org/10.1016/j.nmd.2017.09.006
Messina, Sonia ; Pane, Marika ; Sansone, Valeria ; Bruno, Claudio ; Catteruccia, Michela ; Vita, Giuseppe ; Palermo, Concetta ; Albamonte, Emilio ; Pedemonte, Marina ; Bertini, Enrico ; Binetti, Luca ; Mercuri, Eugenio ; Lauro, Daniela ; Pallara, Anita ; Spinoglio, Simona ; Solinas, Maria Letizia ; Zappa, Grazia ; Penno, Francesca ; Ponzanelli, Cristina ; Casiraghi, Jacopo ; Villanova, Marcello ; Tiziano, Danilo ; Mandelli, Anna ; Mastella, Chiara ; Fontana, Alberto ; Ambrosini, Anna ; Leone, Daniela ; de Sanctis, Roberto ; Forcina, Nicola ; Piastra, Marco ; Genovese, Orazio ; Pedicelli, Alessandro ; Sframeli, Maria ; Versaci, Antonio ; Rulli, Imma ; Gitto, Eloisa ; Faraone, Cristina ; La Foresta, Stefania ; Macrì, Maria ; D'Amico, Adele ; Colia, Giulia ; Bonetti, Anna Maria ; Carlesi, Adelina ; Cutrera, Renato ; Chiarini, Maria Beatrice ; Ferretti, Marta ; Garaventa, Alberto ; Montobbio, Giovanni ; Gandolfo, Carlo ; Iurilli, Valentina ; on behalf of the Italian EAP working Group. / Expanded access program with Nusinersen in SMA type I in Italy : Strengths and pitfalls of a successful experience. In: Neuromuscular Disorders. 2017 ; Vol. 27, No. 12. pp. 1084-1086.
@article{2b1ca03ef0d2408f9be93f7e27a9b070,
title = "Expanded access program with Nusinersen in SMA type I in Italy: Strengths and pitfalls of a successful experience",
author = "Sonia Messina and Marika Pane and Valeria Sansone and Claudio Bruno and Michela Catteruccia and Giuseppe Vita and Concetta Palermo and Emilio Albamonte and Marina Pedemonte and Enrico Bertini and Luca Binetti and Eugenio Mercuri and Daniela Lauro and Anita Pallara and Simona Spinoglio and Solinas, {Maria Letizia} and Grazia Zappa and Francesca Penno and Cristina Ponzanelli and Jacopo Casiraghi and Marcello Villanova and Danilo Tiziano and Anna Mandelli and Chiara Mastella and Alberto Fontana and Anna Ambrosini and Daniela Leone and {de Sanctis}, Roberto and Nicola Forcina and Marco Piastra and Orazio Genovese and Alessandro Pedicelli and Maria Sframeli and Antonio Versaci and Imma Rulli and Eloisa Gitto and Cristina Faraone and {La Foresta}, Stefania and Maria Macr{\`i} and Adele D'Amico and Giulia Colia and Bonetti, {Anna Maria} and Adelina Carlesi and Renato Cutrera and Chiarini, {Maria Beatrice} and Marta Ferretti and Alberto Garaventa and Giovanni Montobbio and Carlo Gandolfo and Valentina Iurilli and {on behalf of the Italian EAP working Group}",
year = "2017",
month = "12",
day = "1",
doi = "10.1016/j.nmd.2017.09.006",
language = "English",
volume = "27",
pages = "1084--1086",
journal = "Neuromuscular Disorders",
issn = "0960-8966",
publisher = "Elsevier Ltd",
number = "12",

}

TY - JOUR

T1 - Expanded access program with Nusinersen in SMA type I in Italy

T2 - Strengths and pitfalls of a successful experience

AU - Messina, Sonia

AU - Pane, Marika

AU - Sansone, Valeria

AU - Bruno, Claudio

AU - Catteruccia, Michela

AU - Vita, Giuseppe

AU - Palermo, Concetta

AU - Albamonte, Emilio

AU - Pedemonte, Marina

AU - Bertini, Enrico

AU - Binetti, Luca

AU - Mercuri, Eugenio

AU - Lauro, Daniela

AU - Pallara, Anita

AU - Spinoglio, Simona

AU - Solinas, Maria Letizia

AU - Zappa, Grazia

AU - Penno, Francesca

AU - Ponzanelli, Cristina

AU - Casiraghi, Jacopo

AU - Villanova, Marcello

AU - Tiziano, Danilo

AU - Mandelli, Anna

AU - Mastella, Chiara

AU - Fontana, Alberto

AU - Ambrosini, Anna

AU - Leone, Daniela

AU - de Sanctis, Roberto

AU - Forcina, Nicola

AU - Piastra, Marco

AU - Genovese, Orazio

AU - Pedicelli, Alessandro

AU - Sframeli, Maria

AU - Versaci, Antonio

AU - Rulli, Imma

AU - Gitto, Eloisa

AU - Faraone, Cristina

AU - La Foresta, Stefania

AU - Macrì, Maria

AU - D'Amico, Adele

AU - Colia, Giulia

AU - Bonetti, Anna Maria

AU - Carlesi, Adelina

AU - Cutrera, Renato

AU - Chiarini, Maria Beatrice

AU - Ferretti, Marta

AU - Garaventa, Alberto

AU - Montobbio, Giovanni

AU - Gandolfo, Carlo

AU - Iurilli, Valentina

AU - on behalf of the Italian EAP working Group

PY - 2017/12/1

Y1 - 2017/12/1

UR - http://www.scopus.com/inward/record.url?scp=85033584960&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85033584960&partnerID=8YFLogxK

U2 - 10.1016/j.nmd.2017.09.006

DO - 10.1016/j.nmd.2017.09.006

M3 - Editorial

AN - SCOPUS:85033584960

VL - 27

SP - 1084

EP - 1086

JO - Neuromuscular Disorders

JF - Neuromuscular Disorders

SN - 0960-8966

IS - 12

ER -