A. Reha

Ataluren treatment of patients with nonsense mutation dystrophinopathy

K. Bushby, Richard S. Finkel, Brenda Wong et al. · Muscle & Nerve · 2014 · 426 citations · Full text

Medicine, Genetics, Molecular Genetics +17

The 6‐minute walk test as a new outcome measure in Duchenne muscular dystrophy

Craig M. McDonald, Erik Henricson, Jay J. Han et al. · Muscle & Nerve · 2009 · 390 citations

Medicine, Physical Medicine, Rehabilitation Medicine +17

THE 6‐minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study

Craig M. McDonald, Erik Henricson, Richard T. Abresch et al. · Muscle & Nerve · 2013 · 301 citations · Full text

Medicine, Physical Medicine, Rehabilitation Medicine +17

Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy

Richard S. Finkel, Kevin M. Flanigan, Brenda Wong et al. · PLoS ONE · 2013 · 241 citations · Full text

Medicine, Genetics, Molecular Genetics +16

Ataluren (PTC124) Induces Cystic Fibrosis Transmembrane Conductance Regulator Protein Expression and Activity in Children with Nonsense Mutation Cystic Fibrosis

Isabelle Sermet‐Gaudelus, K. De Boeck, Georges Casimir et al. · American Journal of Respiratory and Critical Care Medicine · 2010 · 240 citations

Medicine, Pediatrics, Pharmacology +16