Improvements in health status and utility associated with ataluren for the treatment of nonsense mutation Duchenne muscular dystrophy

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

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Health-related quality of life in patients with Duchenne muscular dystrophy: a multinational, cross-sectional study

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Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries

(2015) Todd Gammie et al.   PLoS One

Caregiver Burden

(2014) Ronald D. Adelman et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Ataluren treatment of patients with nonsense mutation dystrophinopathy

(2014) Katharine Bushby et al.   MUSCLE & NERVE

Newborn bloodspot screening for Duchenne Muscular Dystrophy: 21 years experience in Wales (UK)

(2013) Stuart J Moat et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

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

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Evidence-based path to newborn screening for duchenne muscular dystrophy

(2012) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD

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Prevalence of genetic muscle disease in Northern England: in-depth analysis of a muscle clinic population

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