Muscular Dystrophy, Duchenne
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Subject Areas on Research
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A 1.3 kb promoter fragment confers spatial and temporal expression of utrophin A mRNA in mouse skeletal muscle fibers.
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A Transition Toolkit for Duchenne Muscular Dystrophy.
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A step toward the future of seamless measurement with wearable sensors in pediatric populations with neuromuscular diseases.
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AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice.
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AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice.
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Acute heart failure during spinal surgery in a boy with Duchenne muscular dystrophy.
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An ankyrin-based mechanism for functional organization of dystrophin and dystroglycan.
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An unusual cause of dyspnoea in a young man.
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Ataluren treatment of patients with nonsense mutation dystrophinopathy.
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Breathing in Duchenne muscular dystrophy: translation to therapy.
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Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models.
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Cathelicidin-related antimicrobial peptide mediates skeletal muscle degeneration caused by injury and Duchenne muscular dystrophy in mice.
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Clinical and genetic characterization of manifesting carriers of DMD mutations.
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Commentary on "Progression of Ankle Plantarflexion Contractures and Functional Decline in Duchenne Muscular Dystrophy: Implications for Physical Therapy Management".
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Commentary on "Supported Standing in Boys With Duchenne Muscular Dystrophy".
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Comparison of magnetic resonance feature tracking for strain calculation with harmonic phase imaging analysis.
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Comparison of right and left ventricular function and size in Duchenne muscular dystrophy.
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Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases.
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Deflazacort in Duchenne muscular dystrophy: a comparison of two different protocols.
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Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management.
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Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care.
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Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management.
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Duchenne muscular dystrophy and steroids: pharmacologic treatment in the absence of effective gene therapy.
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Dystrophin immunity in Duchenne's muscular dystrophy.
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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.
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Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial.
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Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
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Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study.
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Exercise and Duchenne muscular dystrophy: where we have been and where we need to go.
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Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy.
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Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.
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Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents.
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Glucocorticoid treatment alleviates dystrophic myofiber pathology by activation of the calcineurin/NF-AT pathway.
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Glucocorticoid treatment for the prevention of scoliosis in children with Duchenne muscular dystrophy: long-term follow-up.
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High-Pressure Transvenous Perfusion of the Upper Extremity in Human Muscular Dystrophy: A Safety Study with 0.9% Saline.
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In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.
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Increased expression of utrophin in a slow vs. a fast muscle involves posttranscriptional events.
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Inefficient dystrophin expression after cord blood transplantation in Duchenne muscular dystrophy.
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Lifeline. An interview of Kate Bushby.
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Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.
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Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade.
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Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy.
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Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy.
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Molecular, cellular, and pharmacological therapies for Duchenne/Becker muscular dystrophies.
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Motor axonopathies in a mouse model of Duchenne muscular dystrophy.
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Multiple regulatory events controlling the expression and localization of utrophin in skeletal muscle fibers: insights into a therapeutic strategy for Duchenne muscular dystrophy.
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Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.
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Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy: A Randomized Clinical Trial.
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Myocardial Fibrosis in Duchenne and Becker Muscular Dystrophy-Reply.
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Orthopedic and Surgical Management of the Patient With Duchenne Muscular Dystrophy.
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Patients' and caregivers' maximum acceptable risk of death for non-curative gene therapy to treat Duchenne muscular dystrophy.
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Patterns of left ventricular remodeling in patients with Duchenne Muscular Dystrophy: a cardiac MRI study of ventricular geometry, global function, and strain.
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Pharmacological activation of PPARbeta/delta stimulates utrophin A expression in skeletal muscle fibers and restores sarcolemmal integrity in mature mdx mice.
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Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector.
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Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug.
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Presence of mechanical dyssynchrony in Duchenne muscular dystrophy.
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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.
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Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best-worst scaling experiment in caregivers and adult patients.
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Profoundly lower muscle mass and rate of contractile protein synthesis in boys with Duchenne muscular dystrophy.
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Progress in therapy for Duchenne muscular dystrophy.
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Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients.
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Regional circumferential strain is a biomarker for disease severity in duchenne muscular dystrophy heart disease: a cross-sectional study.
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Rehabilitation Management of the Patient With Duchenne Muscular Dystrophy.
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Relationship of Right Ventricular Size and Function with Respiratory Status in Duchenne Muscular Dystrophy.
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Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial.
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Sarcolemmal nNOS anchoring reveals a qualitative difference between dystrophin and utrophin.
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Serum biomarkers associated with baseline clinical severity in young steroid-naïve Duchenne muscular dystrophy boys.
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Should foot surgery be performed for children with Duchenne muscular dystrophy?
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Sildenafil does not improve cardiomyopathy in Duchenne/Becker muscular dystrophy.
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Steroid treatment and the development of scoliosis in males with duchenne muscular dystrophy.
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Stimulation of calcineurin signaling attenuates the dystrophic pathology in mdx mice.
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Suppression of revertant fibers in mdx mice by expression of a functional dystrophin.
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Targeted inhibition of Ca2+ /calmodulin signaling exacerbates the dystrophic phenotype in mdx mouse muscle.
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The CINRG Becker Natural History Study: Baseline characteristics.
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The Canadian experience with long-term deflazacort treatment in Duchenne muscular dystrophy.
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The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID-19 pandemic.
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The inflammatory pathology of dysferlinopathy is distinct from calpainopathy, Becker muscular dystrophy, and inflammatory myopathies.
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The role of corticosteroids in Duchenne muscular dystrophy: a review for the anesthetist.
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Ultra-widefield imaging of Duchenne muscular dystrophy-associated proliferative retinal vasculopathy improved with panretinal laser photocoagulation alone.
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Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function.
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Keywords of People