Concepedia

European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene

Manuel Haas, Viktor Vlcek, Pavel Balabanov, Tomas Salmonson, Serge Bakchine, Greg Markey, Martina Weise, Gabriele Schlosser‐Weber, Henning Brohmann, Concepción Prieto Yerro,

Neuromuscular Disorders · 2014 · 158 citations · 11 references

DOI

Concepts

References

11

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

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Ataluren treatment of patients with nonsense mutation dystrophinopathy

K. Bushby, Richard S. Finkel, Brenda Wong et al. · Muscle & Nerve · 2014 · 416 citations · Full text

The 6‐minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study

Craig M. McDonald, Erik Henricson, Richard T. Abresch et al. · Muscle & Nerve · 2013 · 335 citations · Full text

Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial

Eitan Kerem, Michael W. Konstan, K. De Boeck et al. · The Lancet Respiratory Medicine · 2014 · 333 citations · Full text

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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 · 295 citations · Full text