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
Early treatment with lisinopril and spironolactone preserves cardiac and skeletal muscle in Duchenne muscular dystrophy mice. Circulation (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
Examination of effects of corticosteroids on skeletal muscles of boys with DMD using MRI and MRS. Neurology (2014) 1.79
LTBP4 genotype predicts age of ambulatory loss in Duchenne muscular dystrophy. Ann Neurol (2013) 1.63
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
Percent-predicted 6-minute walk distance in duchenne muscular dystrophy to account for maturational influences. PLoS Curr (2012) 1.48
Improving recognition of Duchenne muscular dystrophy: a retrospective case note review. Arch Dis Child (2014) 1.46
Health-related quality of life in patients with Duchenne muscular dystrophy: a multinational, cross-sectional study. Dev Med Child Neurol (2015) 1.42
Ataluren treatment of patients with nonsense mutation dystrophinopathy. Muscle Nerve (2014) 1.41
Discovery of Metabolic Biomarkers for Duchenne Muscular Dystrophy within a Natural History Study. PLoS One (2016) 1.39
Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy. Proc Natl Acad Sci U S A (2015) 1.37
Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy. Mol Ther (2011) 1.30
Ataluren as an agent for therapeutic nonsense suppression. Annu Rev Med (2012) 1.24
Lower leg muscle involvement in Duchenne muscular dystrophy: an MR imaging and spectroscopy study. Skeletal Radiol (2011) 1.23
Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124). J Child Neurol (2010) 1.23
Skeletal muscles of ambulant children with Duchenne muscular dystrophy: validation of multicenter study of evaluation with MR imaging and MR spectroscopy. Radiology (2013) 1.22
Mechanical derivation of functional myotubes from adipose-derived stem cells. Biomaterials (2011) 1.22
The burden of Duchenne muscular dystrophy: an international, cross-sectional study. Neurology (2014) 1.21
Newborn bloodspot screening for Duchenne muscular dystrophy: 21 years experience in Wales (UK). Eur J Hum Genet (2013) 1.20
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
Affinity proteomics within rare diseases: a BIO-NMD study for blood biomarkers of muscular dystrophies. EMBO Mol Med (2014) 1.14
Metabolic remodeling agents show beneficial effects in the dystrophin-deficient mdx mouse model. Skelet Muscle (2012) 1.10
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
Magnetic resonance imaging and spectroscopy assessment of lower extremity skeletal muscles in boys with Duchenne muscular dystrophy: a multicenter cross sectional study. PLoS One (2014) 1.07
Tadalafil alleviates muscle ischemia in patients with Becker muscular dystrophy. Sci Transl Med (2012) 1.06
Sulforaphane Attenuates Muscle Inflammation in Dystrophin-deficient mdx Mice via NF-E2-related Factor 2 (Nrf2)-mediated Inhibition of NF-κB Signaling Pathway. J Biol Chem (2015) 1.05
Chemical shift-based MRI to measure fat fractions in dystrophic skeletal muscle. Magn Reson Med (2013) 1.05
Worsening of cardiomyopathy using deflazacort in an animal model rescued by gene therapy. PLoS One (2011) 1.05
Discovery of serum protein biomarkers in the mdx mouse model and cross-species comparison to Duchenne muscular dystrophy patients. Hum Mol Genet (2014) 1.05
O(2) regulates skeletal muscle progenitor differentiation through phosphatidylinositol 3-kinase/AKT signaling. Mol Cell Biol (2011) 1.03
Prednisolone attenuates improvement of cardiac and skeletal contractile function and histopathology by lisinopril and spironolactone in the mdx mouse model of Duchenne muscular dystrophy. PLoS One (2014) 1.01
Genetic modifiers of ambulation in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study. Ann Neurol (2015) 1.01
Validation of genetic modifiers for Duchenne muscular dystrophy: a multicentre study assessing SPP1 and LTBP4 variants. J Neurol Neurosurg Psychiatry (2014) 1.00
Drug treatment of Duchenne muscular dystrophy: available evidence and perspectives. Acta Myol (2012) 0.98
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
Patterns of decline in upper limb function of boys and men with DMD: an international survey. J Neurol (2014) 0.97
Can outcomes in Duchenne muscular dystrophy be improved by public reporting of data? Neurology (2013) 0.97
Natural history of pulmonary function in collagen VI-related myopathies. Brain (2013) 0.97
Enalapril treatment discloses an early role of angiotensin II in inflammation- and oxidative stress-related muscle damage in dystrophic mdx mice. Pharmacol Res (2011) 0.96
Assessing functional performance in the mdx mouse model. J Vis Exp (2014) 0.96
Abnormal circumferential strain is present in young Duchenne muscular dystrophy patients. Pediatr Cardiol (2013) 0.96
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
Incidence and severity of myofiber branching with regeneration and aging. Skelet Muscle (2014) 0.94
Pre-clinical drug tests in the mdx mouse as a model of dystrophinopathies: an overview. Acta Myol (2012) 0.94
Targeted next-generation sequencing as a comprehensive test for patients with and female carriers of DMD/BMD: a multi-population diagnostic study. Eur J Hum Genet (2013) 0.94
Aminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophy. Ther Adv Neurol Disord (2010) 0.93
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy. J Cell Biol (2014) 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
Prednisolone improves walking in Japanese Duchenne muscular dystrophy patients. J Neurol (2013) 0.91
Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: results of a double-blind randomized clinical trial. Neuromuscul Disord (2013) 0.91
Electrical impedance myography for the assessment of children with muscular dystrophy: a preliminary study. J Phys Conf Ser (2013) 0.90
RNAseq analysis for the diagnosis of muscular dystrophy. Ann Clin Transl Neurol (2015) 0.90
Upper extremity 3-dimensional reachable workspace analysis in dystrophinopathy using Kinect. Muscle Nerve (2015) 0.90
Permanent muscular sodium overload and persistent muscle edema in Duchenne muscular dystrophy: a possible contributor of progressive muscle degeneration. J Neurol (2012) 0.90
Compliance to Care Guidelines for Duchenne Muscular Dystrophy. J Neuromuscul Dis (2016) 0.90
Optical coherence tomography can assess skeletal muscle tissue from mouse models of muscular dystrophy by parametric imaging of the attenuation coefficient. Biomed Opt Express (2014) 0.89
Epigallocatechin-3-gallate: a useful, effective and safe clinical approach for targeted prevention and individualised treatment of neurological diseases? EPMA J (2013) 0.89
Treatment with a nitric oxide-donating NSAID alleviates functional muscle ischemia in the mouse model of Duchenne muscular dystrophy. PLoS One (2012) 0.89
Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy. Biomed Res Int (2013) 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
Autophagy is Impaired in the Tibialis Anterior of Dystrophin Null Mice. PLoS Curr (2013) 0.88
Dystrobrevin increases dystrophin's binding to the dystrophin-glycoprotein complex and provides protection during cardiac stress. J Mol Cell Cardiol (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
Pharmacologic management of Duchenne muscular dystrophy: target identification and preclinical trials. ILAR J (2014) 0.88
A mixed methods study of age at diagnosis and diagnostic odyssey for Duchenne muscular dystrophy. Eur J Hum Genet (2015) 0.88
Improving translational studies: lessons from rare neuromuscular diseases. Dis Model Mech (2015) 0.87
Resilience in children diagnosed with a chronic neuromuscular disorder. J Dev Behav Pediatr (2011) 0.87
Imaging deep skeletal muscle structure using a high-sensitivity ultrathin side-viewing optical coherence tomography needle probe. Biomed Opt Express (2013) 0.87
Proteomics reveals drastic increase of extracellular matrix proteins collagen and dermatopontin in the aged mdx diaphragm model of Duchenne muscular dystrophy. Int J Mol Med (2012) 0.87
Effectiveness of diffusion tensor imaging in assessing disease severity in Duchenne muscular dystrophy: preliminary study. Pediatr Radiol (2014) 0.86
Dystropathology increases energy expenditure and protein turnover in the mdx mouse model of duchenne muscular dystrophy. PLoS One (2014) 0.86
Recent developments in the treatment of Duchenne muscular dystrophy and spinal muscular atrophy. Ther Adv Neurol Disord (2013) 0.86
MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy. Gene Ther (2012) 0.86
Burden, professional support, and social network in families of children and young adults with muscular dystrophies. Muscle Nerve (2015) 0.86
The role of oxidative stress in skeletal muscle injury and regeneration: focus on antioxidant enzymes. J Muscle Res Cell Motil (2016) 0.85
Murine and human myogenic cells identified by elevated aldehyde dehydrogenase activity: implications for muscle regeneration and repair. PLoS One (2011) 0.85
Evidence of Insulin Resistance and Other Metabolic Alterations in Boys with Duchenne or Becker Muscular Dystrophy. Int J Endocrinol (2015) 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
Lung function monitoring in patients with duchenne muscular dystrophy on steroid therapy. BMC Res Notes (2012) 0.84
Comparative proteomic profiling of soleus, extensor digitorum longus, flexor digitorum brevis and interosseus muscles from the mdx mouse model of Duchenne muscular dystrophy. Int J Mol Med (2013) 0.84
Evaluation of the therapeutic utility of phosphodiesterase 5A inhibition in the mdx mouse model of duchenne muscular dystrophy. Handb Exp Pharmacol (2011) 0.84
Congenital and childhood myotonic dystrophy: Current aspects of disease and future directions. World J Clin Pediatr (2015) 0.84
Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol (2009) 4.27
Agalsidase-beta therapy for advanced Fabry disease: a randomized trial. Ann Intern Med (2006) 3.38
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
Development of the Performance of the Upper Limb module for Duchenne muscular dystrophy. Dev Med Child Neurol (2013) 1.30
Health-related quality of life in children and adolescents with Duchenne muscular dystrophy. Pediatrics (2012) 1.28
The muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet): surveillance methodology. Birth Defects Res A Clin Mol Teratol (2006) 1.24
Severity, type, and distribution of myotonic discharges are different in type 1 and type 2 myotonic dystrophy. Muscle Nerve (2007) 1.23
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
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A multicomponent behavioral program for oral aversion in children dependent on gastrostomy feedings. J Pediatr Gastroenterol Nutr (2003) 1.08
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Use of corticosteroids in a population-based cohort of boys with duchenne and becker muscular dystrophy. J Child Neurol (2010) 1.04
CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy. Ann Neurol (2005) 1.04
Association of Duchenne muscular dystrophy with autism spectrum disorder. J Child Neurol (2005) 1.04
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
Bone development: interaction of molecular components and biophysical forces. Clin Orthop Relat Res (2005) 0.94
Posterior spinal fusion for scoliosis in duchenne muscular dystrophy diminishes the rate of respiratory decline. Spine (Phila Pa 1976) (2007) 0.94
Assessing upper limb function in nonambulant SMA patients: development of a new module. Neuromuscul Disord (2011) 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
The natural history of cardiac and pulmonary function decline in patients with duchenne muscular dystrophy. Spine (Phila Pa 1976) (2011) 0.91
Leisure characteristics of the pediatric spinal cord injury population. J Spinal Cord Med (2004) 0.91
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The prevalence and impact of scoliosis in Pompe disease: lessons learned from the Pompe Registry. Mol Genet Metab (2011) 0.89
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Variable phenotypes associated with mutations in DOK7. Muscle Nerve (2008) 0.89