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J Pediatr Orthop. 2004 Jan-Feb;24(1):97-101.

Musculoskeletal manifestations of Hurler syndrome: long-term follow-up after bone marrow transplantation.

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Department of Orthopaedic Surgery, University of California, San Francisco, San Francisco, California, USA.


Bone marrow transplantation (BMT) is effective in ameliorating many of the clinical manifestations of Hurler syndrome. However, long-term data on the natural history of the musculoskeletal disorders of Hurler syndrome after BMT are limited. The authors report the orthopaedic outcomes in seven patients with Hurler syndrome who were successfully engrafted between 1990 and 1999, and have been followed for a mean of 7.6 years since transplantation. Medical records, clinical examinations, and imaging studies were reviewed to assess the development and management of hip dysplasia, genu valgum, spine abnormalities, hand abnormalities, and joint range of motion. BMT does not appear to alter the natural history of the musculoskeletal disorders in Hurler syndrome, although there may be a beneficial effect on upper extremity joint mobility.

[Indexed for MEDLINE]

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