Published in Lancet Neurol on November 27, 2009
Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR-DMD) | NCT01603407
The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study. Muscle Nerve (2013) 2.47
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. Mol Ther (2011) 2.06
Targeting RNA to treat neuromuscular disease. Nat Rev Drug Discov (2011) 1.89
Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy. Neurology (2011) 1.86
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. Muscle Nerve (2013) 1.63
Characterization of pulmonary function in Duchenne Muscular Dystrophy. Pediatr Pulmonol (2015) 1.58
Contemporary cardiac issues in Duchenne muscular dystrophy. Working Group of the National Heart, Lung, and Blood Institute in collaboration with Parent Project Muscular Dystrophy. Circulation (2015) 1.55
Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy. Ann Neurol (2016) 1.53
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. Muscle Nerve (2013) 1.48
Poloxamer [corrected] 188 has a deleterious effect on dystrophic skeletal muscle function. PLoS One (2014) 1.46
Supported Standing in Boys With Duchenne Muscular Dystrophy. Pediatr Phys Ther (2016) 1.39
Serial casting for the management of ankle contracture in Duchenne muscular dystrophy. Pediatr Phys Ther (2011) 1.38
Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124). J Child Neurol (2010) 1.23
The burden of Duchenne muscular dystrophy: an international, cross-sectional study. Neurology (2014) 1.21
Prevalence and distribution of late gadolinium enhancement in a large population of patients with Duchenne muscular dystrophy: effect of age and left ventricular systolic function. J Cardiovasc Magn Reson (2013) 1.18
Therapeutic advances in muscular dystrophy. Ann Neurol (2013) 1.17
Participation and quality of life in children with Duchenne muscular dystrophy using the International Classification of Functioning, Disability, and Health. Health Qual Life Outcomes (2012) 1.12
The TREAT-NMD care and trial site registry: an online registry to facilitate clinical research for neuromuscular diseases. Orphanet J Rare Dis (2013) 1.09
Tadalafil alleviates muscle ischemia in patients with Becker muscular dystrophy. Sci Transl Med (2012) 1.06
Chemical shift-based MRI to measure fat fractions in dystrophic skeletal muscle. Magn Reson Med (2013) 1.05
Genetic modifiers of ambulation in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study. Ann Neurol (2015) 1.01
Novel approaches to corticosteroid treatment in Duchenne muscular dystrophy. Phys Med Rehabil Clin N Am (2012) 0.98
Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study. Neurology (2015) 0.98
Can outcomes in Duchenne muscular dystrophy be improved by public reporting of data? Neurology (2013) 0.97
DuchenneConnect Registry Report. PLoS Curr (2012) 0.96
Cooperative International Neuromuscular Research Group Duchenne Natural History Study demonstrates insufficient diagnosis and treatment of cardiomyopathy in Duchenne muscular dystrophy. Muscle Nerve (2014) 0.95
Splicing therapy for neuromuscular disease. Mol Cell Neurosci (2013) 0.95
The Canadian experience with long-term deflazacort treatment in Duchenne muscular dystrophy. Acta Myol (2012) 0.95
Upper limb strength and function changes during a one-year follow-up in non-ambulant patients with Duchenne Muscular Dystrophy: an observational multicenter trial. PLoS One (2015) 0.94
Jagged 1 Rescues the Duchenne Muscular Dystrophy Phenotype. Cell (2015) 0.92
Assessment and management of respiratory function in patients with Duchenne muscular dystrophy: current and emerging options. Ther Clin Risk Manag (2015) 0.91
PDE5 inhibition alleviates functional muscle ischemia in boys with Duchenne muscular dystrophy. Neurology (2014) 0.91
Compliance to Care Guidelines for Duchenne Muscular Dystrophy. J Neuromuscul Dis (2016) 0.90
Treatment with a nitric oxide-donating NSAID alleviates functional muscle ischemia in the mouse model of Duchenne muscular dystrophy. PLoS One (2012) 0.89
Fluoxetine prevents dystrophic changes in a zebrafish model of Duchenne muscular dystrophy. Hum Mol Genet (2014) 0.88
Effects of steroids and angiotensin converting enzyme inhibition on circumferential strain in boys with Duchenne muscular dystrophy: a cross-sectional and longitudinal study utilizing cardiovascular magnetic resonance. J Cardiovasc Magn Reson (2011) 0.88
The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development. Orphanet J Rare Dis (2015) 0.88
Long term non-invasive ventilation in children: impact on survival and transition to adult care. PLoS One (2015) 0.88
Early right ventricular fibrosis and reduction in biventricular cardiac reserve in the dystrophin-deficient mdx heart. Am J Physiol Heart Circ Physiol (2014) 0.88
Resilience in children diagnosed with a chronic neuromuscular disorder. J Dev Behav Pediatr (2011) 0.87
Voltage-gated ion channel dysfunction precedes cardiomyopathy development in the dystrophic heart. PLoS One (2011) 0.87
Cardiac involvement in Duchenne and Becker muscular dystrophy. World J Cardiol (2015) 0.87
Duchenne muscular dystrophy gene therapy in the canine model. Hum Gene Ther Clin Dev (2015) 0.86
Recent developments in the treatment of Duchenne muscular dystrophy and spinal muscular atrophy. Ther Adv Neurol Disord (2013) 0.86
Corticosteroid Treatments in Males With Duchenne Muscular Dystrophy: Treatment Duration and Time to Loss of Ambulation. J Child Neurol (2014) 0.85
Murine and human myogenic cells identified by elevated aldehyde dehydrogenase activity: implications for muscle regeneration and repair. PLoS One (2011) 0.85
Elusive sources of variability of dystrophin rescue by exon skipping. Skelet Muscle (2015) 0.85
Reliability and validity of the Chinese version of the Pediatric Quality Of Life InventoryTM (PedsQLTM) 3.0 neuromuscular module in children with Duchenne muscular dystrophy. Health Qual Life Outcomes (2013) 0.84
Women seeking second opinion for symptomatic uterine leiomyoma: role of comprehensive fibroid center. J Ther Ultrasound (2014) 0.84
Lung function monitoring in patients with duchenne muscular dystrophy on steroid therapy. BMC Res Notes (2012) 0.84
Congenital and childhood myotonic dystrophy: Current aspects of disease and future directions. World J Clin Pediatr (2015) 0.84
Current and emerging treatment strategies for Duchenne muscular dystrophy. Neuropsychiatr Dis Treat (2016) 0.83
Adult care for Duchenne muscular dystrophy in the UK. J Neurol (2014) 0.83
Limb-girdle and congenital muscular dystrophies: current diagnostics, management, and emerging technologies. Curr Neurol Neurosci Rep (2010) 0.83
Extending the translational potential of targeting NO/cGMP-regulated pathways in the CVS. Br J Pharmacol (2015) 0.83
The importance of genetic diagnosis for Duchenne muscular dystrophy. J Med Genet (2016) 0.83
Galectin-1 Protein Therapy Prevents Pathology and Improves Muscle Function in the mdx Mouse Model of Duchenne Muscular Dystrophy. Mol Ther (2015) 0.82
Clinic-based infant screening for duchenne muscular dystrophy: a feasibility study. PLoS Curr (2012) 0.82
Comparative cost of illness analysis and assessment of health care burden of Duchenne and Becker muscular dystrophies in Germany. Orphanet J Rare Dis (2014) 0.82
Selective modulation through the glucocorticoid receptor ameliorates muscle pathology in mdx mice. J Pathol (2013) 0.82
The role of the neuromuscular medicine and physiatry specialists in the multidisciplinary management of neuromuscular disease. Phys Med Rehabil Clin N Am (2012) 0.82
Benefits of glucocorticoids in non-ambulant boys/men with Duchenne muscular dystrophy: A multicentric longitudinal study using the Performance of Upper Limb test. Neuromuscul Disord (2015) 0.82
Transforming growth factor-beta (TGF- β) signaling in paravertebral muscles in juvenile and adolescent idiopathic scoliosis. Biomed Res Int (2014) 0.82
Improving clinical trial design for Duchenne muscular dystrophy. BMC Neurol (2015) 0.81
A comprehensive database of Duchenne and Becker muscular dystrophy patients (0-18 years old) in East China. Orphanet J Rare Dis (2015) 0.81
Long-term ventilation of patients with Duchenne muscular dystrophy: experiences at the Neuromuscular Centre Ulm. Acta Myol (2012) 0.81
A novel in vitro model for studying quiescence and activation of primary isolated human myoblasts. PLoS One (2013) 0.81
Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy. Neurology (2016) 0.81
Dysphagia in Duchenne muscular dystrophy: practical recommendations to guide management. Disabil Rehabil (2016) 0.81
Current Translational Research and Murine Models For Duchenne Muscular Dystrophy. J Neuromuscul Dis (2016) 0.80
Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials. Am J Transl Res (2016) 0.80
Longitudinal Evaluation of Muscle Composition Using Magnetic Resonance in 4 Boys With Duchenne Muscular Dystrophy: Case Series. Phys Ther (2015) 0.80
Autologous bone marrow mononuclear cell transplantation in Duchenne muscular dystrophy - a case report. Am J Case Rep (2014) 0.80
The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials. J Neurol Neurosurg Psychiatry (2015) 0.80
Bone health and associated metabolic complications in neuromuscular diseases. Phys Med Rehabil Clin N Am (2012) 0.80
Alterations of dystrophin-associated glycoproteins in the heart lacking dystrophin or dystrophin and utrophin. J Muscle Res Cell Motil (2013) 0.80
Peptide Nucleic Acid Promotes Systemic Dystrophin Expression and Functional Rescue in Dystrophin-deficient mdx Mice. Mol Ther Nucleic Acids (2015) 0.80
Evaluation of Echocardiographic Measures of Left Ventricular Function in Patients with Duchenne Muscular Dystrophy: Assessment of Reproducibility and Comparison to Cardiac Magnetic Resonance Imaging. J Am Soc Echocardiogr (2016) 0.80
Functional muscle ischemia in Duchenne and Becker muscular dystrophy. Front Physiol (2013) 0.79
Targeting TGF-β Signaling by Antisense Oligonucleotide-mediated Knockdown of TGF-β Type I Receptor. Mol Ther Nucleic Acids (2014) 0.79
Nutritional practices at a glance: spinal muscular atrophy type I nutrition survey findings. J Child Neurol (2013) 0.79
Ambulatory Monitoring and Arrhythmic Outcomes in Pediatric and Adolescent Patients With Duchenne Muscular Dystrophy. J Am Heart Assoc (2015) 0.79
piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts. Nucleic Acids Res (2015) 0.79
Enhanced currents through L-type calcium channels in cardiomyocytes disturb the electrophysiology of the dystrophic heart. Am J Physiol Heart Circ Physiol (2013) 0.78
Nutrition strategies to improve physical capabilities in Duchenne muscular dystrophy. Phys Med Rehabil Clin N Am (2011) 0.78
Current Progress in Therapeutic Gene Editing for Monogenic Diseases. Mol Ther (2016) 0.78
Label-free mass spectrometric analysis reveals complex changes in the brain proteome from the mdx-4cv mouse model of Duchenne muscular dystrophy. Clin Proteomics (2015) 0.78
Early Detection of Myocardial Bioenergetic Deficits: A 9.4 Tesla Complete Non Invasive 31P MR Spectroscopy Study in Mice with Muscular Dystrophy. PLoS One (2015) 0.78
Treatment effect of idebenone on inspiratory function in patients with Duchenne muscular dystrophy. Pediatr Pulmonol (2016) 0.78
Variables associated with upper extremity function in patients with Duchenne muscular dystrophy. J Neurol (2016) 0.77
Motor physical therapy affects muscle collagen type I and decreases gait speed in dystrophin-deficient dogs. PLoS One (2014) 0.77
Quantifying the burden of caregiving in Duchenne muscular dystrophy. J Neurol (2016) 0.77
Comparison of Experimental Protocols of Physical Exercise for mdx Mice and Duchenne Muscular Dystrophy Patients. J Neuromuscul Dis (2015) 0.77
Quality improvement in neurology: muscular dystrophy quality measures. Neurology (2015) 0.77
DMD genotypes and loss of ambulation in the CINRG Duchenne Natural History Study. Neurology (2016) 0.77
Disparities in health care utilization by race among teenagers and young adults with muscular dystrophy. Med Care (2014) 0.77
Upper Limb Evaluation in Duchenne Muscular Dystrophy: Fat-Water Quantification by MRI, Muscle Force and Function Define Endpoints for Clinical Trials. PLoS One (2016) 0.76
Quantitative analysis of the reversibility of knee flexion contractures with time: an experimental study using the rat model. BMC Musculoskelet Disord (2014) 0.76
Age-related longitudinal changes in metabolic energy expenditure during walking in boys with Duchenne muscular dystrophy. PLoS One (2014) 0.76
Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol (2009) 8.42
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A randomized study of alglucosidase alfa in late-onset Pompe's disease. N Engl J Med (2010) 3.05
Pompe disease diagnosis and management guideline. Genet Med (2006) 3.03
A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophy. Ann Neurol (2008) 2.95
Delayed diagnosis in duchenne muscular dystrophy: data from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet). J Pediatr (2009) 2.90
Gastrointestinal microflora studies in late-onset autism. Clin Infect Dis (2002) 2.80
Glycogen storage disease type III diagnosis and management guidelines. Genet Med (2010) 2.13
Corticosteroid treatment retards development of ventricular dysfunction in Duchenne muscular dystrophy. Neuromuscul Disord (2008) 2.10
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. Mol Ther (2011) 2.06
Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease. Genet Med (2012) 2.05
ICU-Acquired Weakness Is Associated With Differences in Clinical Outcomes in Critically Ill Children. Pediatr Crit Care Med (2016) 2.02
NF-κB inhibition delays DNA damage-induced senescence and aging in mice. J Clin Invest (2012) 1.91
Coordinate interaction between IL-13 and epithelial differentiation cluster genes in eosinophilic esophagitis. J Immunol (2010) 1.85
A striking local esophageal cytokine expression profile in eosinophilic esophagitis. J Allergy Clin Immunol (2011) 1.82
LTBP4 genotype predicts age of ambulatory loss in Duchenne muscular dystrophy. Ann Neurol (2013) 1.63
Glucocorticoid-regulated genes in eosinophilic esophagitis: a role for FKBP51. J Allergy Clin Immunol (2010) 1.63
The impact of antibodies in late-onset Pompe disease: a case series and literature review. Mol Genet Metab (2012) 1.60
Limb-girdle muscular dystrophy in the United States. J Neuropathol Exp Neurol (2006) 1.59
Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa. Mol Genet Metab (2012) 1.58
Role of innate immunity in a murine model of histidyl-transfer RNA synthetase (Jo-1)-mediated myositis. Arthritis Rheum (2011) 1.56
Statement on the care of the child with chronic lung disease of infancy and childhood. Am J Respir Crit Care Med (2003) 1.54
Molecular diagnosis of eosinophilic esophagitis by gene expression profiling. Gastroenterology (2013) 1.51
Widening gap in age at muscular dystrophy-associated death between blacks and whites, 1986-2005. Neurology (2010) 1.50
An enteric-coated high-buffered pancrelipase reduces steatorrhea in patients with cystic fibrosis: a prospective, randomized study. J Am Diet Assoc (2006) 1.50
Skeletal development in fetal pig specimens: MR imaging of femur with histologic comparison. Radiology (2004) 1.49
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. Muscle Nerve (2013) 1.48
Pompe disease: design, methodology, and early findings from the Pompe Registry. Mol Genet Metab (2011) 1.48
Change in natural history of Duchenne muscular dystrophy with long-term corticosteroid treatment: implications for management. J Child Neurol (2010) 1.44
Tranexamic acid reduces intraoperative blood loss in pediatric patients undergoing scoliosis surgery. Anesthesiology (2005) 1.43
Liquid formulation of pentoxifylline is a poorly tolerated treatment for duchenne dystrophy. Muscle Nerve (2011) 1.42
Ataluren treatment of patients with nonsense mutation dystrophinopathy. Muscle Nerve (2014) 1.41
Ordering molecular genetic tests and reporting results: practices in laboratory and clinical settings. J Mol Diagn (2008) 1.40
The emerging phenotype of long-term survivors with infantile Pompe disease. Genet Med (2012) 1.39
Structural stages in the development of the long bones and epiphyses: a study in the New Zealand white rabbit. J Bone Joint Surg Am (2002) 1.38
Observational study of spinal muscular atrophy type 2 and 3: functional outcomes over 1 year. Arch Neurol (2011) 1.32
Eosinophilic esophagitis in infants and toddlers. Dysphagia (2006) 1.31
DMD Trp3X nonsense mutation associated with a founder effect in North American families with mild Becker muscular dystrophy. Neuromuscul Disord (2009) 1.31
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Splicing biomarkers of disease severity in myotonic dystrophy. Ann Neurol (2013) 1.22
Corticosteroids in Duchenne muscular dystrophy: major variations in practice. Muscle Nerve (2013) 1.20
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Peptide-based inhibition of NF-κB rescues diaphragm muscle contractile dysfunction in a murine model of Duchenne muscular dystrophy. Mol Med (2011) 1.09
Rapamycin ameliorates dystrophic phenotype in mdx mouse skeletal muscle. Mol Med (2011) 1.09
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CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy. Ann Neurol (2005) 1.04
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Computerized hand grip myometry reliably measures myotonia and muscle strength in myotonic dystrophy (DM1). Muscle Nerve (2007) 1.03
Oral corticosteroids and onset of cardiomyopathy in Duchenne muscular dystrophy. J Pediatr (2013) 1.02
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Sternal bands for closure of midline sternotomy leads to better wound healing. Asian Cardiovasc Thorac Ann (2007) 0.95
Cooperative International Neuromuscular Research Group Duchenne Natural History Study demonstrates insufficient diagnosis and treatment of cardiomyopathy in Duchenne muscular dystrophy. Muscle Nerve (2014) 0.95
Severe respiratory syncytial virus pneumonia associated with primary Epstein-Barr virus infection. Pediatr Pulmonol (2002) 0.95
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Assessing upper limb function in nonambulant SMA patients: development of a new module. Neuromuscul Disord (2011) 0.94
Posterior spinal fusion for scoliosis in duchenne muscular dystrophy diminishes the rate of respiratory decline. Spine (Phila Pa 1976) (2007) 0.94
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Mutation analysis in a population-based cohort of boys with Duchenne or Becker muscular dystrophy. J Child Neurol (2008) 0.93
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Systemic delivery of NEMO binding domain/IKKγ inhibitory peptide to young mdx mice improves dystrophic skeletal muscle histopathology. Neurobiol Dis (2011) 0.92
MicroRNA-486-dependent modulation of DOCK3/PTEN/AKT signaling pathways improves muscular dystrophy-associated symptoms. J Clin Invest (2014) 0.92
Leisure characteristics of the pediatric spinal cord injury population. J Spinal Cord Med (2004) 0.91
The natural history of cardiac and pulmonary function decline in patients with duchenne muscular dystrophy. Spine (Phila Pa 1976) (2011) 0.91
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Variable phenotypes associated with mutations in DOK7. Muscle Nerve (2008) 0.89