M. Garralda

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

J. Bourke, K. Bushby, F. Muntoni, 2011, The Lancet.

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.

K. Bushby, F. Muntoni, K. Anthony, 2011, Lancet.

Emotional impact of genetic trials in progressive paediatric disorders: a dose-ranging exon-skipping trial in Duchenne muscular dystrophy.

A. Couteur, H. McConachie, K. Bushby, 2013, Child: care, health and development.

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

I. Graham, K. Bushby, F. Muntoni, 2009, The Lancet Neurology.

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

V. Arechavala-Gomeza, S. Çırak, M. Guglieri, 2011, The Lancet.

Emotional impact of genetic trials in progressive paediatric disorders: a dose-ranging exon-skipping trial in Duchenne muscular dystrophy.

A. Couteur, H. McConachie, K. Bushby, 2013, Child: care, health and development.

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

I. Graham, K. Bushby, L. Popplewell, 2009, The Lancet Neurology.

Siblings of young people with Duchenne muscular dystrophy--a qualitative study of impact and coping.

F. Muntoni, M. Kinali, T. Weaver, 2011, European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society.