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Abstract
We report on the case of a girl with type 1 Gaucher’s disease, treated from age 9 to 15 with high-dose enzyme replacement therapy. This treatment did not avert the development of an extensive mutilating hepatic fibrosis warranting a liver transplantation, which was followed by death.
In some cases of Gaucher’s disease, alternative strategies such as fractionated or further increased ERT, gene therapy, or glucosyltransferase inhibitor should be explored.
- Received March 14, 2001.
- Accepted November 19, 2001.
- Copyright © 2002 by the American Academy of Pediatrics
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