title: Duchenne and Becker muscular dystrophies.
abstract: The dystrophinopathies Duchenne and Becker muscular dystrophies (DMD and BMD) represent the most common inherited disorders of muscle. Improvements in cardiac care, attention to respiratory function, and judicious use of spinal correction surgery have led to increased survival in the DMD population. Meanwhile, advances in molecular therapeutics have led to promising therapies that are in or are entering clinical trials. An understanding of the dystrophinopathies, and recent advances in their molecular diagnosis and treatment, is of benefit to practicing neurologists.
authors.affiliation: Center for Gene Therapy, Room 3014, The Research Institute of Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43209, USA. Electronic address: kevin.flanigan@nationwidechildrens.org.
authors.forename: Kevin M
authors.initials: KM
authors.lastname: Flanigan
mesh_terms: D016189:Dystrophin / Q000235:genetics / Q000378:metabolism; D006801:Humans; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000175:diagnosis* / Q000235:genetics / Q000378:metabolism / Q000628:therapy*
chemical_list: D016189:Dystrophin
keywords: Becker muscular dystrophy; Duchenne muscular dystrophy; Dystrophin; Exon skipping