Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. / Cirak, Sebahattin; Arechavala-Gomeza, Virginia; Guglieri, Michela; Feng, Lucy; Torelli, Silvia; Anthony, Karen; Abbs, Stephen; Garralda, Maria Elena; Bourke, John; Wells, Dominic J.; Dickson, George; Wood, Matthew J. A.; Wilton, Steve D.; Straub, Volker; Kole, Ryszard; Shrewsbury, Stephen B.; Sewry, Caroline; Morgan, Jennifer E.; Bushby, Kate; Muntoni, Francesco.

In: The Lancet, Vol. 378, No. 9791, 13.08.2011, p. 595-605.

Research output: Contribution to journalArticle

Published

  • Sebahattin Cirak
  • Virginia Arechavala-Gomeza
  • Michela Guglieri
  • Lucy Feng
  • Silvia Torelli
  • Karen Anthony
  • Stephen Abbs
  • Maria Elena Garralda
  • John Bourke
  • Dominic J. Wells
  • George Dickson
  • Matthew J. A. Wood
  • Steve D. Wilton
  • Volker Straub
  • Ryszard Kole
  • Stephen B. Shrewsbury
  • Caroline Sewry
  • Jennifer E. Morgan
  • Kate Bushby
  • Francesco Muntoni
Original languageEnglish
Pages595-605
Number of pages11
JournalThe Lancet
Journal publication date13 Aug 2011
Journal number9791
Volume378
Early online date25/07/11
DOIs
StatePublished

ID: 4081292