The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review

Genetic modifiers of ambulation in the cooperative international Neuromuscular research group Duchenne natural history study

(2015) Luca Bello et al.   ANNALS OF NEUROLOGY

Prevalence of Fatigue, Pain, and Affective Disorders in Adults With Duchenne Muscular Dystrophy and Their Associations With Quality of Life

(2015) Robert F. Pangalila et al.   ARCHIVES OF PHYSICAL MEDICINE AND REHABILITATION

Is functional dependence of Duchenne muscular dystrophy patients determinant of the quality of life and burden of their caregivers?

(2015) Maria Clara Drummond Soares de Moura et al.   ARQUIVOS DE NEURO-PSIQUIATRIA

Socio-economic burden of rare diseases: A systematic review of cost of illness evidence

(2015) Aris Angelis et al.   HEALTH POLICY

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

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

Characterization of pulmonary function in Duchenne Muscular Dystrophy

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

Prevalence of Duchenne and Becker Muscular Dystrophies in the United States

(2015) P. A. Romitti et al.   PEDIATRICS

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

Left Ventricular Dysfunction in Duchenne Muscular Dystrophy and Genotype

(2014) Mahi L. Ashwath et al.   AMERICAN JOURNAL OF CARDIOLOGY

A Population-Based Study of Dystrophin Mutations in Canada

(2014) Jean K. Mah et al.   CANADIAN JOURNAL OF NEUROLOGICAL SCIENCES

Participation in daily life activities and its relationship to strength and functional measures in boys with Duchenne muscular dystrophy

(2014) Roxanna M. Bendixen et al.   DISABILITY AND REHABILITATION

Observations of body mass index in Duchenne muscular dystrophy: a longitudinal study

(2014) Z E Davidson et al.   EUROPEAN JOURNAL OF CLINICAL NUTRITION

Trends With Corticosteroid Use in Males With Duchenne Muscular Dystrophy Born 1982-2001

(2014) Deborah J. Fox et al.   JOURNAL OF CHILD NEUROLOGY

Adult care for Duchenne muscular dystrophy in the UK

(2014) Sunil Rodger et al.   JOURNAL OF NEUROLOGY

Patterns of decline in upper limb function of boys and men with DMD: an international survey

(2014) Mariska M. H. P. Janssen et al.   JOURNAL OF NEUROLOGY

Cooperative international neuromuscular research group duchenne natural history study demonstrates insufficient diagnosis and treatment of cardiomyopathy in duchenne muscular dystrophy

(2014) Christopher Spurney et al.   MUSCLE & NERVE

Prevalence of Muscular Dystrophies: A Systematic Literature Review

(2014) Alice Theadom et al.   NEUROEPIDEMIOLOGY

The burden of Duchenne muscular dystrophy: An international, cross-sectional study

(2014) E. Landfeldt et al.   NEUROLOGY

A systematic review and meta-analysis on the epidemiology of Duchenne and Becker muscular dystrophy

(2014) Jean K. Mah et al.   NEUROMUSCULAR DISORDERS

Comparative cost of illness analysis and assessment of health care burden of Duchenne and Becker muscular dystrophies in Germany

(2014) Olivia Schreiber-Katz et al.   Orphanet Journal of Rare Diseases

6 Minute Walk Test in Duchenne MD Patients with Different Mutations: 12 Month Changes

(2014) Marika Pane 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

The level of agreement between child self-reports and parent proxy-reports of health-related quality of life in boys with Duchenne muscular dystrophy

(2014) Yoonjeong Lim et al.   QUALITY OF LIFE RESEARCH

Health Related Quality of Life of Patients and Their Caregivers In Rare Diseases Results of the Burqol-Rd Project In Hungary

(2014) M. Péntek et al.   VALUE IN HEALTH

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

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

Growth and psychomotor development of patients with Duchenne muscular dystrophy

(2013) Elisabeth Sarrazin et al.   EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY

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

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

Health-Related Quality of Life and Its Relation to Disease Severity in Boys With Duchenne Muscular Dystrophy

(2013) S. L. S. Houwen–van Opstal et al.   JOURNAL OF CHILD NEUROLOGY

Patterns of Growth in Ambulatory Males with Duchenne Muscular Dystrophy

(2013) Nancy A. West et al.   JOURNAL OF PEDIATRICS

Muscular dystrophies

(2013) Eugenio Mercuri et al.   LANCET

The cooperative international neuromuscular research group duchenne natural history study-a longitudinal investigation in the era of glucocorticoid therapy: Design of protocol and the methods used

(2013) Craig M. McDonald 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

Cost of illness for neuromuscular diseases in the United States

(2013) Jane Larkindale 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

Reliability of the walking energy cost test and the six-minute walk test in boys with Duchenne muscular dystrophy

(2013) J.C.E. Kempen 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

Respiratory muscle decline in duchenne muscular dystrophy

(2013) Sonia Khirani et al.   PEDIATRIC PULMONOLOGY

Correction: 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

Specific profiles of neurocognitive and reading functions in a sample of 42 Italian boys with Duchenne Muscular Dystrophy

(2012) Maria Luisa Lorusso et al.   CHILD NEUROPSYCHOLOGY

Participation and quality of life in children with Duchenne muscular dystrophy using the international classification of functioning, disability, and health

(2012) Roxanna M Bendixen et al.   Health and Quality of Life Outcomes

Correlation of Heart Rate and Cardiac Dysfunction in Duchenne Muscular Dystrophy

(2012) Tamara O. Thomas et al.   PEDIATRIC CARDIOLOGY

Health-Related Quality of Life in Children and Adolescents With Duchenne Muscular Dystrophy

(2012) K. Uzark et al.   PEDIATRICS

Bone mass development in patients with Duchenne and Becker muscular dystrophies: a 4-year clinical follow-up

(2011) Ann-Charlott Söderpalm et al.   ACTA PAEDIATRICA

Duchenne muscular dystrophy: quality of life among 95 patients evaluated using the Life Satisfaction Index for Adolescents

(2011) Valdecir A. Simon et al.   ARQUIVOS DE NEURO-PSIQUIATRIA

Clinical and molecular characterization of a cohort of patients with novel nucleotide alterations of the Dystrophin gene detected by direct sequencing

(2011) Francesca Magri et al.   BMC Medical Genetics

Natural evolution of weight status in Duchenne muscular dystrophy: a retrospective audit

(2011) Léonie Martigne et al.   BRITISH JOURNAL OF NUTRITION

Oral Presentations

(2011)   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Quality of Life in Duchenne Muscular Dystrophy: The Subjective Impact on Children and Parents

(2011) Ilaria Baiardini et al.   JOURNAL OF CHILD NEUROLOGY

Genotype and phenotype characterization in a large dystrophinopathic cohort with extended follow-up

(2011) Francesca Magri et al.   JOURNAL OF NEUROLOGY

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

(2011) E. Mazzone et al.   NEUROLOGY

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

(2011) Rolando Roberto et al.   SPINE

Increased catalase expression improves muscle function inmdxmice

(2010) Joshua T. Selsby   EXPERIMENTAL PHYSIOLOGY

Relationship Between Clinical Outcome Measures and Parent Proxy Reports of Health-Related Quality of Life in Ambulatory Children With Duchenne Muscular Dystrophy

(2010) Craig M. McDonald et al.   JOURNAL OF CHILD NEUROLOGY

Tobramycin inhalation powder for P. aeruginosa infection in cystic fibrosis: The EVOLVE trial

(2010) Michael W. Konstan et al.   PEDIATRIC PULMONOLOGY

The respiratory management of patients with duchenne muscular dystrophy: A DMD care considerations working group specialty article

(2010) David J. Birnkrant et al.   PEDIATRIC PULMONOLOGY

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

(2009) Fiona L. M. Norwood et al.   BRAIN

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

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

The Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) statement: guidelines for reporting observational studies

(2008) Erik von Elm et al.   JOURNAL OF CLINICAL EPIDEMIOLOGY