Recent advances in the management of Duchenne muscular dystrophy.
Arch Dis Child
; 100(12): 1173-7, 2015 Dec.
Article
em En
| MEDLINE
| ID: mdl-26153505
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 â¼25â
years. This progress has been made possible through advances in the diagnosis, treatment and long-term care of patients with DMD. 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 boys with DMD about recent scientific advances affecting their child's clinical care.
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Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Assistência de Longa Duração
/
Distrofia Muscular de Duchenne
Idioma:
En
Ano de publicação:
2015
Tipo de documento:
Article