References
- Neufeld, E. F. and Muenzer, J.: The mucopolysaccharidoses. In: C. R. Scrive, A. L. Beaudet, W. S. Sly and D. Valle (editors), The Metabolic and Molecular Bases of Inherited Disease, 7th ed., vol. 2 (New York: McGraw-Hill), pp. 2465–2494, 1995.
- Terlato, N. J. and Cox, G. F.: Can mucopolysaccharidosis type I disease severity be predicted based on a patient’s genotype? A comprehensive review of the literature. Genetics in Medicine, 5: 286–294, 2003.
- Vellodi, A., Young, E. P., Cooper, A. et al. : Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres. Archives of Diseases in Childhood, 76: 92–99, 1997.
- Peters, C., Shapiro, E. G., Anderson, J. et al. : Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group. Blood, 91: 2601–2608, 1998.
- Wenger, D. A., Coppola, S. and Liu, S. L.: Insights into the diagnosis and treatment of lysosomal storage diseases. Archives of Neurology, 60: 322–328, 2003.
- Kakkis, E. D., Muenzer, J., Tiller, G. E. et al. : Enzyme- replacement therapy in mucopolysaccharidosis I. New England Journal of Medicine, 18: 182–188, 2001.
- Kakavanos, R., Turner, C. T., Hopwood, J. J. et al. : Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I. Lancet, 361: 1608–1613, 2003.
- van der Schouw, Y. T., van den Beld, W. A., van der Sanden, G. A. C. et al. : Item assessment in the development of a diagnostic motor performance test for myopathy in children. Developmental Medicine & Child Neurology, 35: 608–613, 1993.
- Haley, S. M., Coster, W. J., Ludlow, L. H. et al. : Pediatric Evaluation of Disability Inventory: Development, Standardization and Administration Manual (Boston, MA: Trustees of Boston University), 1992.
- Podsiadlo, D. and Richardson, S.: The timed ‘Up & Go’: a test of basic functional mobility for frail elderly persons. Journal of the American Geriatric Society, 39: 142–148, 1991.
- Bruininks Oseretsky Test of Motor Proficiency (Circle Pines, MN: American Guidance Service), 1978.
- Russell, D. J., Rosenbaum, P. L., Avery, L. M. et al. : Gross Motor Function Measure (GMFM-66 and GMFM-88) User’s Manual (Cambridge: Cambridge University Press), 2002.
- Brach, J. S., Van Swearingen, J. M., Newman, A. B. et al. : Identifying early decline of physical function in community-dwelling older women: performance-based and self-report measures. Physical Therapy, 82: 320–328, 2002.
- Alexander, N. B., Grunwalt, J. C., Carlos, S. et al. : Bed mobility task performance in older adults. Journal of Rehabilitation Research and Development, 37: 633–638, 2000.
- Triffit, P. D.: The relationship between motion of the shoulder and the stated ability to perform activities of daily living. Journal of Bone and Joint Surgery, 80: 41–47, 1998.
- Engelberg, R., Martin, D. P., Agel, J. et al. : Musculoskeletal function assessment instrument: criterion and construct validity. Journal of Orthopedic Research, 14: 182–192, 1996.
- Daltroy, K. H., Liang, M. H., Fossel, A. H. et al. : The POSNA pediatric musculoskeletal functional health questionnaire: report on reliability, validity and sensitivity to change. Journal of Pediatric Orthopedics, 18: 561–571, 1998.
- Rose, J., Gamble, J. G., Medeiros, J. et al. : Energy cost of walking in normal children and in those with cerebral palsy: comparison of heart rate and oxygen uptake. Journal of Pediatric Orthopedics, 9: 276–279, 1989.
- Portney, L. G. and Watkins, M. P.: Foundations of Clinical Research: Applications to Practice, 2nd ed. (Boston, MA: Prentice-Hall), 2000.
- Goodgold, S., Corcoran, M., Gamache, D. et al. : Backpack use in children. Pediatric Physical Therapy, 14: 122–131, 2002.