title: The muscular dystrophies.
abstract: The muscular dystrophies are disorders of progressive muscular degeneration and weakness. As a group they display clinical heterogeneity that reflects the heterogeneity of molecular mechanisms responsible for them, and range from congenital to adulthood onset. Recent advances in the field include improved methods of diagnosis, continued identification of disease genes, and the development of a unified model of pathogenesis in facioscapulohumeral dystrophy. These advances are reflected in the development of new therapeutic approaches, some of which have already led to clinical trials in the dystrophinopathies and limb-girdle dystrophies.
authors.affiliation: Departments of Pediatrics and Neurology, The Center for Gene Therapy, The Research Institute, Nationwide Children's Hospital, The Ohio State University, Columbus, Ohio 43205, USA. kevin.flanigan@nationwidechildrens.org
authors.forename: Kevin M
authors.initials: KM
authors.lastname: Flanigan
mesh_terms: D000305:Adrenal Cortex Hormones / Q000627:therapeutic use; D000818:Animals; D000893:Anti-Inflammatory Agents / Q000627:therapeutic use; D016189:Dystrophin / Q000235:genetics; D006801:Humans; D009136:Muscular Dystrophies / Q000151:congenital / Q000188:drug therapy / Q000235:genetics / Q000628:therapy*; D049288:Muscular Dystrophies, Limb-Girdle / Q000188:drug therapy / Q000235:genetics; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy / Q000235:genetics; D020391:Muscular Dystrophy, Facioscapulohumeral / Q000188:drug therapy / Q000235:genetics
chemical_list: D000305:Adrenal Cortex Hormones; D000893:Anti-Inflammatory Agents; C484258:DMD protein, human; D016189:Dystrophin