用于球形细胞脑白质营养不良、肾上腺脑白质营养不良、异染性脑白质营养不良和Hurler综合征的骨髓移植。
Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome.
作者信息
Krivit W, Aubourg P, Shapiro E, Peters C
机构信息
University of Minnesota and Institute of Human Genetics, Minneapolis 55455, USA.
出版信息
Curr Opin Hematol. 1999 Nov;6(6):377-82. doi: 10.1097/00062752-199911000-00004.
Abstract
Bone marrow transplantation protocols for inherited metabolic storage diseases are unique for each disorder treated. Differences depend also upon how old the patient was when onset occurred and rate of progression of disease. Treatment is directed to prevent or ameliorate the inexorable neurological deterioration that is the major pathophysiological event in all of these inherited metabolic storage diseases.
摘要
针对遗传性代谢性贮积病的骨髓移植方案，对于所治疗的每种疾病而言都是独特的。差异还取决于发病时患者的年龄以及疾病的进展速度。治疗旨在预防或改善不可避免的神经功能恶化，而这种恶化是所有这些遗传性代谢性贮积病的主要病理生理事件。
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