Comparison of Mutation Profiles in the Duchenne Muscular Dystrophy Gene among Populations: Implications for Potential Molecular Therapies

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

(2015) Manuel Haas et al.   NEUROMUSCULAR DISORDERS

Ataluren: First Global Approval

(2014) Nicola J. Ryan   DRUGS

Antisense-mediated exon skipping: Taking advantage of a trick from Mother Nature to treat rare genetic diseases

(2014) Marcel Veltrop et al.   EXPERIMENTAL CELL RESEARCH

A novel noncontiguous duplication in the DMD gene escapes the ‘reading-frame rule’

(2014) LUZ BERENICE LÓPEZ-HERNÁNDEZ et al.   JOURNAL OF GENETICS

Ataluren treatment of patients with nonsense mutation dystrophinopathy

(2014) Katharine Bushby et al.   MUSCLE & NERVE

Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice

(2014) Nicolas Wein et al.   NATURE MEDICINE

The TREAT-NMD Duchenne Muscular Dystrophy Registries: Conception, Design, and Utilization by Industry and Academia

(2013) Catherine L. Bladen et al.   HUMAN MUTATION

Mutation Spectrum ofDystrophinGene in Malaysian Patients with Duchenne/Becker Muscular Dystrophy

(2013) Abdul Qawee Rani et al.   JOURNAL OF NEUROGENETICS

Exon Deletion Patterns of the Dystrophin Gene in 82 Vietnamese Duchenne/Becker Muscular Dystrophy Patients

(2013) Van Khanh Tran et al.   JOURNAL OF NEUROGENETICS

194th ENMC international workshop. 3rd ENMC workshop on exon skipping: Towards clinical application of antisense-mediated exon skipping for Duchenne muscular dystrophy 8–10 December 2012, Naarden, The Netherlands

(2013) Annemieke Aartsma-Rus et al.   NEUROMUSCULAR DISORDERS

Characteristics of Japanese Duchenne and Becker muscular dystrophy patients in a novel Japanese national registry of muscular dystrophy (Remudy)

(2013) Harumasa Nakamura et al.   Orphanet Journal of Rare Diseases

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

(2013) Richard S. Finkel et al.   PLoS One

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

(2011) Nathalie M. Goemans et al.   NEW ENGLAND JOURNAL OF MEDICINE

Change in Natural History of Duchenne Muscular Dystrophy With Long-term Corticosteroid Treatment: Implications for Management

(2010) Richard T. Moxley et al.   JOURNAL OF CHILD NEUROLOGY

Pre-Hispanic Mesoamerican demography approximates the present-day ancestry of Mestizos throughout the territory of Mexico

(2009) Rodrigo Rubi-Castellanos et al.   AMERICAN JOURNAL OF PHYSICAL ANTHROPOLOGY

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

(2009) Annemieke Aartsma-Rus et al.   HUMAN MUTATION

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

(2009) Maria Kinali et al.   LANCET NEUROLOGY

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

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

Rapid and cost effective detection of small mutations in the DMD gene by high resolution melting curve analysis

(2009) Rowida Almomani et al.   NEUROMUSCULAR DISORDERS

Similarity of DMD gene deletion and duplication in the Chinese patients compared to global populations

(2008) Xiaozhu Wang et al.   Behavioral and Brain Functions

Intragenic deletions in the dystrophin gene in 211 Pakistani Duchenne muscular dystrophy patients

(2008) Muhammad J Hassan et al.   PEDIATRICS INTERNATIONAL