Evaluation of serum MMP-9 as predictive biomarker for antisense therapy in Duchenne

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

K. Bushby, Richard S. Finkel, David J. Birnkrant et al. · The Lancet Neurology · 2009 · 2K citations

Part 1, Kinesiology, Psychiatry +9

PTC124 targets genetic disorders caused by nonsense mutations

Ellen Welch, Elisabeth R. Barton, Jin Zhuo et al. · Nature · 2007 · 1.1K citations

Mendelian Disorder, Genetic Disorder, Genetics +4

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 · 852 citations · Full text

Exon Skipping, Urology, Duchenne Muscular Dystrophy +4

Functional improvement of dystrophic muscle by myostatin blockade

Sasha Bogdanovich, Thomas Krag, Elisabeth R. Barton et al. · Nature · 2002 · 819 citations

Muscle Function, Skeletal Muscle, Physiology +2

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 · 760 citations