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

1. Cirak, S.
2. Arechavala-Gomeza, V.
3. Guglieri, M.
4. Feng, L.
5. Torelli, S.
6. Anthony, K.
7. Abbs, S.
8. Garralda, M.E.
9. Bourke, J.
10. Wells, D.J.
11. Dickson, G.
12. Wood, M.J.
13. Wilton, S.D.
14. Straub, V.
15. Kole, R.
16. Shrewsbury, S.B.
17. Sewry, C.
18. Morgan, J.E.
19. Bushby, K.
20. Muntoni, F.