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A scale to monitor progression and treatment of mitochondrial disease in children.
- Source :
-
Neuromuscular disorders : NMD [Neuromuscul Disord] 2006 Dec; Vol. 16 (12), pp. 814-20. Date of Electronic Publication: 2006 Nov 22. - Publication Year :
- 2006
-
Abstract
- Mitochondrial diseases affect all age groups, but those with childhood onset often seem to experience the greatest burden of disability. In some paediatric patients this can be explained by a cumulative disability acquired over many years. In others, additional factors, including the nature and severity of the molecular defect, must be considered. To date, no large-scale studies have attempted to document the natural history of paediatric mitochondrial disease. This is in part at least, because no assessment tool has been available to plot the temporal course of a disease with such a diverse clinical spectrum. This paper describes how a practical and semi-quantitative rating scale has been devised for children with mitochondrial disease, the Newcastle paediatric mitochondrial disease scale (NPMDS). The scale is multi-dimensional and reproducible, offering a tool through which mitochondrial disease progression can be objectively monitored. We anticipate that use of this tool will facilitate both longitudinal natural history studies and the assessment of future therapeutic interventions.
- Subjects :
- Adolescent
Child
Child, Preschool
Disease Progression
Female
Humans
Infant
Infant, Newborn
Male
Neurology methods
Observer Variation
Pediatrics methods
Predictive Value of Tests
Prognosis
Reproducibility of Results
United Kingdom
Disability Evaluation
Mitochondrial Diseases diagnosis
Mitochondrial Diseases therapy
Mitochondrial Encephalomyopathies diagnosis
Mitochondrial Encephalomyopathies therapy
Subjects
Details
- Language :
- English
- ISSN :
- 0960-8966
- Volume :
- 16
- Issue :
- 12
- Database :
- MEDLINE
- Journal :
- Neuromuscular disorders : NMD
- Publication Type :
- Academic Journal
- Accession number :
- 17123819
- Full Text :
- https://doi.org/10.1016/j.nmd.2006.08.006