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Treatment of Duchenne and Becker muscular dystrophy

Author
Basil T Darras, MD
Section Editor
Marc C Patterson, MD, FRACP
Deputy Editor
John F Dashe, MD, PhD

INTRODUCTION

The muscular dystrophies are an inherited group of progressive myopathic disorders resulting from defects in a number of genes required for normal muscle function [1]. The Duchenne and Becker muscular dystrophies (as well as a third intermediate form) are caused by mutations of the dystrophin gene and are therefore named dystrophinopathies. Weakness is the principal symptom as muscle fiber degeneration is the primary pathologic process.

The dystrophinopathies are inherited as X-linked recessive traits and have varying clinical characteristics:

Duchenne muscular dystrophy (DMD) is associated with the most severe clinical symptoms.

Becker muscular dystrophy (BMD) has a similar presentation to DMD but a relatively milder clinical course.

An intermediate group of patients, known as "outliers," may be classified clinically as having either mild DMD or severe BMD.

                               

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Literature review current through: Nov 2016. | This topic last updated: Thu Oct 27 00:00:00 GMT+00:00 2016.
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