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Recent advances in the management of Duchenne muscular dystrophy

Lookup NU author(s): Dr Eugen-Matthias Strehle, Professor Volker StraubORCiD

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Abstract

Duchenne muscular dystrophy (DMD) is the commonest inherited neuromuscular disorder of childhood and mainly affects males. Over the course of the last century the average life expectancy of these patients has doubled and now stands at ~25 years. This progress has been made possible through advances in the diagnosis, treatment and long-term care of DMD patients. Basic and clinical research, national and international scientific networks, and parent and patient support groups have all contributed to achieving this goal. The advent of molecular genetic therapies and personalised medicine has opened up new avenues and raised hopes that one day a cure for this debilitating orphan disease will be found. The main purpose of this short review is to enable paediatricians to have informed discussions with parents of DMD boys about recent scientific advances affecting their child’s clinical care.

Publication metadata

Author(s): Strehle EM, Straub V

Publication type: Review

Publication status: Published

Journal: Archives of Disease in Childhood

Year: 2015

Volume: 100

Issue: 12

Pages: 1173-1177

Print publication date: 01/12/2015

Online publication date: 07/07/2015

Acceptance date: 09/06/2015

ISSN (print): 0003-9888

ISSN (electronic): 1468-2044

URL: http://dx.doi.org/10.1136/archdischild-2014-307962

DOI: 10.1136/archdischild-2014-307962

PubMed id: 26153505

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