Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy

A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy

(2018) Nathalie Goemans et al.   NEUROMUSCULAR DISORDERS

Injection site reactions after subcutaneous oligonucleotide therapy

(2016) Leonie van Meer et al.   BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

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

(2016) Nathalie M. Goemans et al.   PLoS One

The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations

(2015) Catherine L. Bladen et al.   HUMAN MUTATION

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

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

(2014) Marika Pane et al.   PLoS One

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

The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and othe

(2013) Erik K. Henricson et al.   MUSCLE & NERVE

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

(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

24 Month Longitudinal Data in Ambulant Boys with Duchenne Muscular Dystrophy

(2013) Elena Stacy Mazzone et al.   PLoS One

Evidence-based path to newborn screening for duchenne muscular dystrophy

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

Quantitative muscle strength assessment in duchenne muscular dystrophy: longitudinal study and correlation with functional measures

(2012) Alberto Lerario et al.   BMC Neurology

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

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

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

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

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

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

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

(2009) Craig M. McDonald et al.   MUSCLE & NERVE