Common therapeutic advances for Duchenne muscular dystrophy (DMD)

Source: International Journal of Neuroscience - Category: Neuroscience Authors: Source Type: research

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Muscular dystrophies are debilitating disorders that result in progressive weakness and degeneration of skeletal muscle. Although the genetic mutations and clinical abnormalities of a variety of neuromuscular diseases are well known, no curative therapies have been developed to date. The advent of genome editing technology provides new opportunities to correct the underlying mutations responsible for many monogenic neuromuscular diseases. For example, Duchenne muscular dystrophy, which is caused by mutations in the dystrophin gene, has been successfully corrected in mice, dogs, and human cells through CRISPR/Cas9 editing. ...
Source: Journal of Clinical Investigation - Category: Biomedical Science Authors: Source Type: research
Abstract Professor Andrew John Lees, from the National Hospital for Neurology and Neurosurgery, a neurological hospital in Queen Square, London, UK, has contributed in a stupendous way to the development of the field of movement disorders in Brazil, with a constant and intense participation in numerous congresses and scientific meetings of this specialty since 1983.Resumo O professor Andrew Lees, do National Hospital for Neurology and Neurosurgery, Queen Square, Londres, Reino Unido, tem contribu ído de maneira estupenda para o desenvolvimento da área dos distúrbios do movimento no Brasil, com uma part...
Source: Arquivos de Neuro-Psiquiatria - Category: Neurology Source Type: research
Publication date: August 2020Source: Pharmacological Research, Volume 158Author(s): Brigida Boccanegra, Ingrid E.C. Verhaart, Ornella Cappellari, Elizabeth Vroom, Annamaria De Luca
Source: Pharmacological Research - Category: Drugs & Pharmacology Source Type: research
AbstractCardiomyopathy is the leading cause of death in Duchenne muscular dystrophy (DMD). Elevated troponin levels are observed in DMD and may vary with disease progression. We studied troponin levels in DMD related to cardiac fibrosis and native T1 measures. This is a prospective, cross-sectional, observational study of 30 DMD subjects measuring native T1 levels and late gadolinium enhancement (LGE) on cardiac MR imaging (CMR) correlated with temporally associated serum troponin I levels. Non-parametric analyses including Spearman correlations and Kruskal –Wallis test were performed between groups.pvalues resulting...
Source: Mammalian Genome - Category: Genetics & Stem Cells Source Type: research
ObjectiveMuscle inflammation is a feature in myositis and Duchenne muscular dystrophy (DMD ). Autoimmune mechanisms are thought to contribute to muscle weakness in patients with myositis. However, a lack of correlation between the extent of inflammatory cell infiltration and muscle weakness indicates that nonimmune pathologic mechanisms may play a role. The present study focused on 2 microRNA (miRNA ) sets previously identified as being elevated in the muscle of patients with DMD —an “inflammatory” miRNA set that is dampened with glucocorticoids, and a “dystrophin‐targeting” miRNA set that i...
Source: Arthritis and Rheumatology - Category: Rheumatology Authors: Tags: Original Article Source Type: research
(UT Southwestern Medical Center) A joint program of UT Southwestern Medical Center and Children's Health has been approved as a Certified Duchenne Care Center (CDCC) by Parent Project Muscular Dystrophy (PPMD), the nation's most comprehensive nonprofit organization focused on finding a cure for Duchenne muscular dystrophy.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
Varied detection methods have resulted in conflicting reports on the prevalence of cardiac disease in Duchenne and Becker muscular dystrophy carriers (MDC).
Source: International Journal of Cardiology - Category: Cardiology Authors: Source Type: research
Interview with Paula R. Clemens, MD, author of Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial
Source: JAMA Specialty Journals Author Interviews - Category: General Medicine Authors: Source Type: podcasts
This phase 2 randomized clinical trial evaluates the safety, tolerability, and efficacy of viltolarsen in boys with Duchenne muscular dystrophy amenable to exon 53 skipping
Source: JAMA Neurology - Category: Neurology Source Type: research
Publication date: September 2020Source: Molecular Genetics and Metabolism Reports, Volume 24Author(s): Claire Lefeuvre, Stéphane Schaeffer, Robert-Yves Carlier, Maxime Fournier, Françoise Chapon, Valérie Biancalana, Guillaume Nicolas, Edoardo Malfatti, Pascal Laforêt
Source: Molecular Genetics and Metabolism Reports - Category: Genetics & Stem Cells Source Type: research
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