Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome.
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.
Krivit
W
W
University of Minnesota and Institute of Human Genetics, Minneapolis 55455, USA. krivi001@maroon.tc.umn.edu
Aubourg
P
P
Shapiro
E
E
Peters
C
C
D000326:Adrenoleukodystrophy / Q000503:physiopathology / Q000628:therapy*; D016026:Bone Marrow Transplantation; D006801:Humans; D007965:Leukodystrophy, Globoid Cell / Q000503:physiopathology / Q000628:therapy*; D007966:Leukodystrophy, Metachromatic / Q000503:physiopathology / Q000628:therapy*; D008059:Mucopolysaccharidosis I / Q000503:physiopathology / Q000628:therapy*