A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

Clinical Trial ID NCT03439670

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Top papers

Rank Title Journal Year PubWeight™‹?›
1 Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell 1987 20.27
2 Activators and target genes of Rel/NF-kappaB transcription factors. Oncogene 1999 13.46
3 Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol 2009 8.42
4 Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev 2008 3.54
5 Interplay of IKK/NF-kappaB signaling in macrophages and myofibers promotes muscle degeneration in Duchenne muscular dystrophy. J Clin Invest 2007 2.99
6 The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy. Muscle Nerve 2010 2.78
7 Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes. PLoS One 2014 2.43
8 Oral corticosteroids and fracture risk: relationship to daily and cumulative doses. Rheumatology (Oxford) 2000 2.13
9 Reliability of the North Star Ambulatory Assessment in a multicentric setting. Neuromuscul Disord 2009 2.13
10 Eplerenone for early cardiomyopathy in Duchenne muscular dystrophy: a randomised, double-blind, placebo-controlled trial. Lancet Neurol 2014 2.13
11 Mechanical stress activates the nuclear factor-kappaB pathway in skeletal muscle fibers: a possible role in Duchenne muscular dystrophy. FASEB J 2003 2.12
12 Clinical trial in Duchenne dystrophy. I. The design of the protocol. Muscle Nerve 1981 2.06
13 Helper (CD4(+)) and cytotoxic (CD8(+)) T cells promote the pathology of dystrophin-deficient muscle. Clin Immunol 2001 1.94
14 Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy. Neurology 2011 1.86
15 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 other commonly used clinical trial outcome measures. Muscle Nerve 2013 1.55
16 New developments in the pathogenesis and treatment of steroid-induced osteoporosis. J Bone Miner Res 1999 1.54
17 A systematic review and meta-analysis on the epidemiology of Duchenne and Becker muscular dystrophy. Neuromuscul Disord 2014 1.48
18 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
19 Do immune cells promote the pathology of dystrophin-deficient myopathies? Neuromuscul Disord 2001 1.43
20 Regulation of phosphatidylinositol 3-kinase (PI3K)/Akt and nuclear factor-kappa B signaling pathways in dystrophin-deficient skeletal muscle in response to mechanical stretch. J Cell Physiol 2006 1.24
21 Immune-mediated mechanisms potentially regulate the disease time-course of duchenne muscular dystrophy and provide targets for therapeutic intervention. PM R 2009 1.22
22 VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects. EMBO Mol Med 2013 1.22
23 Loss of positive allosteric interactions between neuronal nitric oxide synthase and phosphofructokinase contributes to defects in glycolysis and increased fatigability in muscular dystrophy. Hum Mol Genet 2009 1.15
24 Emerging drugs for Duchenne muscular dystrophy. Expert Opin Emerg Drugs 2012 1.11
25 Tests of adrenal insufficiency. Arch Dis Child 1999 1.11
26 Glucocorticoid-treated mice are an inappropriate positive control for long-term preclinical studies in the mdx mouse. PLoS One 2012 1.10
27 Short term linear growth in asthmatic children during treatment with prednisolone. BMJ 1990 1.06
28 Systemic effects of epidural and intra-articular glucocorticoid injections in diabetic and non-diabetic patients. Joint Bone Spine 2007 1.03
29 Genetic modifiers of ambulation in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study. Ann Neurol 2015 1.01
30 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
31 Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy. J Cell Biol 2014 0.92
32 VBP15: preclinical characterization of a novel anti-inflammatory delta 9,11 steroid. Bioorg Med Chem 2013 0.88
33 VBP15, a novel anti-inflammatory, is effective at reducing the severity of murine experimental autoimmune encephalomyelitis. Cell Mol Neurobiol 2014 0.83
34 Prednisone affects inflammation, glucose tolerance, and bone turnover within hours of treatment in healthy individuals. Eur J Endocrinol 2011 0.82
35 VBP15, a glucocorticoid analogue, is effective at reducing allergic lung inflammation in mice. PLoS One 2013 0.81
36 Changes in blood glucose and cortisol levels after epidural or shoulder intra-articular glucocorticoid injections in diabetic or nondiabetic patients. Am J Phys Med Rehabil 2014 0.81
37 Glucocorticoid effects on statural growth. Br J Rheumatol 1993 0.80
38 Serum pharmacodynamic biomarkers for chronic corticosteroid treatment of children. Sci Rep 2016 0.78
39 Safety and pharmacodynamic dose response of short-term prednisone in healthy adult subjects: a dose ranging, randomized, placebo-controlled, crossover study. BMC Musculoskelet Disord 2016 0.78
40 The effect of alternate-day low dose prednisolone on bone age in children with steroid dependent nephrotic syndrome. Int Urol Nephrol 1997 0.76
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