Format

Send to

Choose Destination
Neurol Clin. 2014 Aug;32(3):671-88, viii. doi: 10.1016/j.ncl.2014.05.002.

Duchenne and Becker muscular dystrophies.

Author information

1
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.

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.

KEYWORDS:

Becker muscular dystrophy; Duchenne muscular dystrophy; Dystrophin; Exon skipping

PMID:
25037084
DOI:
10.1016/j.ncl.2014.05.002
[Indexed for MEDLINE]

Supplemental Content

Full text links

Icon for Elsevier Science
Loading ...
Support Center