Jing Chen, Hua Jiang, Lu Dong, Yaoping Wang, Changying Luo, Min Zhou, Weiming Zhang, Shangzhi Huang, Xuefan Gu, Wenjuan Qiu, Huiwen Zhang, Longjun Gu
OBJECTIVE: Mucopolysaccharidosis(MPS) is a congenital hereditary disease. Only a few patients with this disease can be controlled by enzyme replacement therapy. Most of them are short of effective interference. To exploit the effect of treatment with allogenic hematopoietic stem cell transplantation, two children were treated with the transplantation. METHODS: The two patients included a 23 month MPS-IH and an 18 month old MPS-VI at the time of transplantation. Busulfan of 20 mg/kg plus 200 mg of Cyclophosphamide were used as the conditioning regimen...
December 2008: Zhonghua Yi Xue Yi Chuan Xue za Zhi, Zhonghua Yixue Yichuanxue Zazhi, Chinese Journal of Medical Genetics