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Semin Neurol 2012; 32(03): 255-263
DOI: 10.1055/s-0032-1329199
Thieme Medical Publishers 333 Seventh Avenue, New York, NY 10001, USA.

The Muscular Dystrophies

Kevin M. Flanigan
1   Departments of Pediatrics and Neurology, The Center for Gene Therapy, The Research Institute, Nationwide Children's Hospital, The Ohio State University, Columbus, Ohio
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Publication History

Publication Date:
01 November 2012 (online)

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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.

Keywords

muscular dystrophy - dystrophinopathy - facioscapulohumeral - limb-girdle
 

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