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Published in 2022 at "JAMA Network Open"
DOI: 10.1001/jamanetworkopen.2021.44178
Abstract: This nonrandomized controlled trial examines efficacy of vamorolone treatment for Duchenne muscular dystrophy among boys compared with glucocorticoid treatment.
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Keywords:
efficacy safety;
duchenne muscular;
safety vamorolone;
muscular dystrophy ... See more keywords
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Published in 2018 at "Febs Letters"
DOI: 10.1002/1873-3468.13099
Abstract: Duchenne muscular dystrophy (DMD) is a fatal disorder caused by absence of functional dystrophin protein. Compensation in dystrophin‐deficient (mdx) mice may be achieved by overexpression of its fetal paralogue, utrophin. Strategies to increase utrophin levels…
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Keywords:
mice;
muscular dystrophy;
dystrophin deficient;
duchenne muscular ... See more keywords
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Published in 2021 at "Annals of Clinical and Translational Neurology"
DOI: 10.1002/acn3.51417
Abstract: To understand the natural disease upper limb progression over 3 years of ambulatory and non‐ambulatory patients with Duchenne muscular dystrophy (DMD) using functional assessments and quantitative magnetic resonance imaging (MRI) and to exploratively identify prognostic…
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Keywords:
patients duchenne;
muscular dystrophy;
disease;
upper limb ... See more keywords
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Published in 2022 at "ESC Heart Failure"
DOI: 10.1002/ehf2.13970
Abstract: Despite advances in contemporary cardiopulmonary therapies, cardiomyopathy remains the leading cause of death in patients with Duchenne muscular dystrophy (DMD). Also, the long‐term clinical outcomes of patients with DMD and cardiomyopathy is unknown. This study…
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Keywords:
long term;
clinical outcomes;
duchenne muscular;
patients duchenne ... See more keywords
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Published in 2018 at "Journal of Clinical Laboratory Analysis"
DOI: 10.1002/jcla.22445
Abstract: To offer 4‐year clinical prenatal diagnosis experience of Duchenne muscular dystrophy (DMD).
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Keywords:
prenatal diagnosis;
analysis;
muscular dystrophy;
duchenne muscular ... See more keywords
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Published in 2019 at "Journal of Cachexia, Sarcopenia and Muscle"
DOI: 10.1002/jcsm.12405
Abstract: Muscle wasting and weakness in Duchenne muscular dystrophy (DMD) causes severe locomotor limitations and early death due in part to respiratory muscle failure. Given that current clinical practice focuses on treating secondary complications in this…
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Keywords:
elevated mitochondrial;
muscular dystrophy;
mitochondrial h2o2;
h2o2 emission ... See more keywords
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Published in 2020 at "Journal of Cachexia, Sarcopenia and Muscle"
DOI: 10.1002/jcsm.12569
Abstract: Abstract Background Duchenne muscular dystrophy (DMD) is a progressive muscle‐wasting disease caused by mutations in the dystrophin gene, which leads to structural instability of the dystrophin–glycoprotein‐complex with subsequent muscle degeneration. In addition, muscle inflammation has…
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Keywords:
igg;
muscle;
long term;
treatment ... See more keywords
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Published in 2019 at "Journal of Magnetic Resonance Imaging"
DOI: 10.1002/jmri.26681
Abstract: Duchenne muscular dystrophy (DMD) is a hereditary neuromuscular disease leading to progressive muscle wasting. Since there is a need for MRI variables that serve as early sensitive indicators of response to treatment, several quantitative MRI…
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Keywords:
mri depicts;
muscle;
muscular dystrophy;
23na mri ... See more keywords
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Published in 2021 at "Journal of Magnetic Resonance Imaging"
DOI: 10.1002/jmri.27729
Abstract: Clinical management of boys with Duchenne muscular dystrophy (DMD) relies on in‐depth understanding of cardiac involvement, but right ventricular (RV) structural and functional remodeling remains understudied.
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Keywords:
ventricular function;
boys duchenne;
muscular dystrophy;
right ventricular ... See more keywords
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Published in 2019 at "Molecular Genetics & Genomic Medicine"
DOI: 10.1002/mgg3.622
Abstract: Duchenne muscular dystrophy (DMD) is the most common form of inherited muscular dystrophy. Germline mutations in dystrophin (DMD) gene cause DMD, with a X‐linked recessive mode of inheritance. Patients with DMD are usually characterized by…
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Keywords:
dmd;
muscular dystrophy;
dystrophy;
application whole ... See more keywords
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Published in 2019 at "Molecular Genetics & Genomic Medicine"
DOI: 10.1002/mgg3.862
Abstract: Variable disease phenotypes can be influenced by several factors such as allelic variation, environmental factors, genetic modifiers, and genotype–environment interaction. Herein to the best of our knowledge, this is the first report of the coexistence…
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Keywords:
korean family;
disease phenotypes;
differing disease;
disease ... See more keywords