The development of antisense oligonucleotide therapies for Duchenne muscular dystrophy: Report on a TREAT-NMD workshop hosted by the European Medicines Agency (EMA), on September 25th 2009

Local Dystrophin Restoration with Antisense Oligonucleotide PRO051

Judith C. van Deutekom, Anneke A. M. Janson, Ieke B. Ginjaar et al. · New England Journal of Medicine · 2007 · 762 citations

Medicine, Molecular Genetics, Molecular Medicine +15

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

Changes in Spirometry Over Time as a Prognostic Marker in Patients with Duchenne Muscular Dystrophy

Margaret Phillips, Rosaline C. M. Quinlivan, R. H. T. Edwards et al. · American Journal of Respiratory and Critical Care Medicine · 2001 · 283 citations

Medicine, Physiology, Pulmonary Function Testing +16

The PedsQL™ in pediatric patients with Spinal Muscular Atrophy: Feasibility, reliability, and validity of the Pediatric Quality of Life Inventory™ Generic Core Scales and Neuromuscular Module

Susan T. Iannaccone, Linda S. Hynan, Anne A. Morton et al. · Neuromuscular Disorders · 2009 · 163 citations

Medicine, Child Health, Pediatrics +11

Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy

J. Mayhew, Julaine Florence, Thomas P. Mayhew et al. · Muscle & Nerve · 2006 · 121 citations · Full text

Medicine, Rehabilitation Medicine, Exercise Testing +16