Natural History of Steroid-Treated Young Boys With Duchenne Muscular Dystrophy Using the NSAA, 100m, and Timed Functional Tests

The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy

(2020) Annemieke Aartsma-Rus et al.   Nucleic Acid Therapeutics

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy

(2020) Jerry R. Mendell et al.   JAMA Neurology

Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy

(2019) Anne M. Connolly et al.   MUSCLE & NERVE

Longitudinal natural history in young boys with Duchenne Muscular Dystrophy

(2019) Giorgia Coratti et al.   NEUROMUSCULAR DISORDERS

Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy

(2019) Francesco Muntoni et al.   PLoS One

DMD genotype correlations from the Duchenne Registry: Endogenous exon skipping is a factor in prolonged ambulation for individuals with a defined mutation subtype

(2018) Richard T. Wang et al.   HUMAN MUTATION

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management

(2018) David J Birnkrant et al.   LANCET NEUROLOGY

DUCHENNE MUSCULAR DYSTROPHY - PHYSIOTHERAPY

(2018) N. Miller et al.   NEUROMUSCULAR DISORDERS

The 100-meter timed test: Normative data in healthy males and comparative pilot outcome data for use in Duchenne muscular dystrophy clinical trials

(2017) Lindsay N. Alfano et al.   NEUROMUSCULAR DISORDERS

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

(2016) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Revised North Star Ambulatory Assessment for Young Boys with Duchenne Muscular Dystrophy

(2016) Eugenio Mercuri et al.   PLoS One

Improving clinical trial design for Duchenne muscular dystrophy

(2015) Luciano Merlini et al.   BMC Neurology

Ataluren treatment of patients with nonsense mutation dystrophinopathy

(2014) Katharine Bushby et al.   MUSCLE & NERVE

One Year Outcome of Boys With Duchenne Muscular Dystrophy Using the Bayley-III Scales of Infant and Toddler Development

(2014) Anne M. Connolly et al.   PEDIATRIC NEUROLOGY

Eteplirsen for the treatment of Duchenne muscular dystrophy

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

Detecting meaningful change using the North Star Ambulatory Assessment in Duchenne muscular dystrophy

(2013) Anna G Mayhew et al.   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

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

Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network

(2013) Anne M. Connolly et al.   NEUROMUSCULAR DISORDERS

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

(2011) E. Mazzone et al.   NEUROLOGY

Reliability of the North Star Ambulatory Assessment in a multicentric setting

(2009) E.S. Mazzone et al.   NEUROMUSCULAR DISORDERS