Michela Guglieri

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

Sebahattin Çirak, Virginia Arechavala‐Gomeza, Michela Guglieri et al. · The Lancet · 2011 · 862 citations · Full text

Medicine, Neurology, Pharmacotherapy +13

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

Maria Kinali, Virginia Arechavala‐Gomeza, Lucy Feng et al. · The Lancet Neurology · 2009 · 639 citations · Full text

Life Sciences, Genetics, Molecular Biology +17

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Craig M. McDonald, Craig Campbell, Ricardo Erazo Torricelli et al. · The Lancet · 2017 · 455 citations · Full text

Medicine, Molecular Medicine, Neurology +9

Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy

Diane E. Frank, Frederick J. Schnell, Cody Akana et al. · Neurology · 2020 · 325 citations · Full text

Medicine, Pharmacokinetics, Pharmacotherapy +16

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

Thomas Voit, Haluk Topaloğlu, Volker Straub et al. · The Lancet Neurology · 2014 · 295 citations

Medicine, Pharmacology, Pharmacotherapy +5