Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study

Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial

(2015) Gunnar M Buyse et al.   LANCET

Characterization of pulmonary function in Duchenne Muscular Dystrophy

(2015) O.H. Mayer et al.   PEDIATRIC PULMONOLOGY

Upper Limb Strength and Function Changes during a One-Year Follow-Up in Non-Ambulant Patients with Duchenne Muscular Dystrophy: An Observational Multicenter Trial

(2015) Andreea Mihaela Seferian et al.   PLoS One

Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko mice

(2014) B Wu et al.   GENE THERAPY

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

(2014) Thomas Voit et al.   LANCET NEUROLOGY

A Sensitive, Reproducible and Objective Immunofluorescence Analysis Method of Dystrophin in Individual Fibers in Samples from Patients with Duchenne Muscular Dystrophy

(2014) Chantal Beekman et al.   PLoS One

Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes

(2014) Marika Pane et al.   PLoS One

Eteplirsen for the treatment of Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   MUSCLE & NERVE

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

(2013) Craig M. Mcdonald et al.   MUSCLE & NERVE

195th ENMC International Workshop: Newborn screening for Duchenne muscular dystrophy 14–16th December, 2012, Naarden, The Netherlands

(2013) Juliet A. Ellis et al.   NEUROMUSCULAR DISORDERS

Ambulatory capacity and disease progression as measured by the 6-minute-walk-distance in Duchenne muscular dystrophy subjects on daily corticosteroids

(2013) Nathalie Goemans et al.   NEUROMUSCULAR DISORDERS

Reliability of the Performance of Upper Limb assessment in Duchenne muscular dystrophy

(2013) Marika Pane et al.   NEUROMUSCULAR DISORDERS

Respiratory muscle decline in duchenne muscular dystrophy

(2013) Sonia Khirani et al.   PEDIATRIC PULMONOLOGY

24 Month Longitudinal Data in Ambulant Boys with Duchenne Muscular Dystrophy

(2013) Elena Stacy Mazzone et al.   PLoS One

Innovative methods to assess upper limb strength and function in non-ambulant Duchenne patients

(2012) L. Servais et al.   NEUROMUSCULAR DISORDERS

Restoring Dystrophin Expression in Duchenne Muscular Dystrophy Muscle

(2011) Eric P. Hoffman et al.   AMERICAN JOURNAL OF PATHOLOGY

Functional changes in Duchenne muscular dystrophy: A 12-month longitudinal cohort study

(2011) E. Mazzone et al.   NEUROLOGY

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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

The Natural History of Cardiac and Pulmonary Function Decline in Patients With Duchenne Muscular Dystrophy

(2011) Rolando Roberto et al.   SPINE

The 6-minute walk test in Duchenne/Becker muscular dystrophy: Longitudinal observations

(2010) Craig M. MCDonald et al.   MUSCLE & NERVE

Becker muscular dystrophy patients with deletions around exon 51; a promising outlook for exon skipping therapy in Duchenne patients

(2010) A.T.J.M. Helderman-van den Enden et al.   NEUROMUSCULAR DISORDERS

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

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

Guidelines for Antisense Oligonucleotide Design and Insight Into Splice-modulating Mechanisms

(2008) Annemieke Aartsma-Rus et al.   MOLECULAR THERAPY