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 journal › Article › peer-review
Published
| Original language | English |
|---|---|
| Pages (from-to) | 595-605 |
| Number of pages | 11 |
| Journal | The Lancet |
| Volume | 378 |
| Issue number | 9791 |
| Early online date | 25 Jul 2011 |
| DOIs | |
| Publication status | Published - 13 Aug 2011 |
This open access research output is licenced under a Creative Commons Attribution-NonCommercial-NoDerivs 3.0 Unported License.
ID: 4081292