Hurler syndrome surgery: Difference between revisions
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* These procedures lead to replacement of mutated/ non-functional cells of the bone marrow with functional cells. | * These procedures lead to replacement of mutated/ non-functional cells of the bone marrow with functional cells. | ||
* Functional new cells act as a source for the deficient enzyme. | * Functional new cells act as a source for the deficient enzyme. | ||
* Transplant has the best outcome when its done as soon as the diagnosis is established. | |||
* Children who receive a transplant early enough can have normal or near-normal mental development. | |||
==References== | ==References== |
Revision as of 03:40, 26 February 2013
Hurler Syndrome Microchapters |
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Treatment |
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Hurler syndrome surgery On the Web |
American Roentgen Ray Society Images of Hurler syndrome surgery |
Risk calculators and risk factors for Hurler syndrome surgery |
Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]
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Overview
Bone marrow transplantation (BMT) and umbilical cord blood transplantation (UCBT) can successfully treat the mucopolysaccharidoses. Abnormal physical characteristics, except for those affecting the skeleton and eyes, can be improved, and neurologic degeneration can often be halted. BMT and UCBT are high-risk procedures with high rates of morbidity and mortality. However, they are the only treatments that have the potential to cure the disease.
Surgery
Bone Marrow Transplantation and Umbilical Cord Blood Transplantation
- These procedures lead to replacement of mutated/ non-functional cells of the bone marrow with functional cells.
- Functional new cells act as a source for the deficient enzyme.
- Transplant has the best outcome when its done as soon as the diagnosis is established.
- Children who receive a transplant early enough can have normal or near-normal mental development.