Update on the management of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is familiar to paediatricians as the most common childhood muscular dystrophy and leads to severe disability and early death in the late teenage years if untreated. Improvements in general care, glucocorticoid corticosteroid treatment, non-invasive ventilatory suppo...

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Bibliographic Details
Published in:Archives of disease in childhood 2008-11, Vol.93 (11), p.986-990
Main Authors: Manzur, A Y, Kinali, M, Muntoni, F
Format: Article
Language:English
Subjects:
Adults
Assistive Technology
Biological and medical sciences
Cardiomyopathy, Dilated - etiology
Cardiomyopathy, Dilated - therapy
Care and treatment
Coding
Death
Development and progression
Developmental Delays
Diseases of striated muscles. Neuromuscular diseases
Duchenne muscular dystrophy
Dystrophin - genetics
Dystrophy
Evaluation
Family (Sociological Unit)
Family medical history
General aspects
Genes
Genetic engineering
Genetic Therapy - methods
Genetics
Glucocorticoids - therapeutic use
Health services
Humans
Kinases
Male
Medical sciences
Miscellaneous
Muscular dystrophy
Muscular Dystrophy, Duchenne - complications
Muscular Dystrophy, Duchenne - diagnosis
Muscular Dystrophy, Duchenne - genetics
Muscular Dystrophy, Duchenne - therapy
Mutation
Neurology
Patient Care Team
Patients
Physical Activities
Physical Therapy Modalities
Practice guidelines (Medicine)
Prevention and actions
Proteins
Public health. Hygiene
Public health. Hygiene-occupational medicine
Resistance (Psychology)
Respiration Disorders - etiology
Respiration Disorders - therapy
Rodents
Scoliosis - etiology
Scoliosis - therapy
Screening Tests
Social Services
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Description
Summary:Duchenne muscular dystrophy (DMD) is familiar to paediatricians as the most common childhood muscular dystrophy and leads to severe disability and early death in the late teenage years if untreated. Improvements in general care, glucocorticoid corticosteroid treatment, non-invasive ventilatory support, and cardiomyopathy and scoliosis management have significantly changed the course of DMD in treated individuals, so that survival into adulthood is now a realistic possibility for most patients. This has important implications for the medical and social sectors involved in the transition to adult medical services and the provision of suitable employment and social care. Multidisciplinary team working for optimal management of DMD-specific multisystem complications is essential, and collaboration in disease specific national clinical networks is recommended. Several curative therapeutic strategies including cell and gene therapy are being pursued but are still at an experimental stage.
ISSN:0003-9888
1468-2044
DOI:10.1136/adc.2007.118141