Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome
- 1 November 1999
- journal article
- review article
- Published by Wolters Kluwer Health in Current Opinion in Hematology
- Vol. 6 (6) , 377-82
- https://doi.org/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.Keywords
This publication has 26 references indexed in Scilit:
- Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis, aspartylglucosaminuria, Hurler, Maroteaux-Lamy, and Sly syndromes, and Gaucher disease type IIICurrent Opinion in Neurology, 1999
- The neurobiology of X-linked adrenoleukodystrophy, a demyelinating peroxisomal disorderTrends in Neurosciences, 1999
- Lovastatin for X-Linked AdrenoleukodystrophyNew England Journal of Medicine, 1998
- Lovastatin and sodium phenylacetate normalize the levels of very long chain fatty acids in skin fibroblasts of X‐ adrenoleukodystrophyFEBS Letters, 1998
- Hematopoietic Stem-Cell Transplantation in Globoid-Cell LeukodystrophyNew England Journal of Medicine, 1998
- Adrenoleukodystrophy: phenotype, genetics, pathogenesis and therapyBrain, 1997
- Engraftment following in utero bone marrow transplantation for globoid cell leukodystrophyBone Marrow Transplantation, 1997
- Multislice proton magnetic resonance spectroscopic imaging in X‐linked adrenoleukodystrophyAnnals of Neurology, 1994
- Childhood adrenoleukodystrophy: assessment with proton MR spectroscopy.Radiology, 1993
- Late-Onset Krabbe Disease (Globoid Cell Leukodystrophy): Clinical and Biochemical Features of 15 CasesDevelopmental Neuroscience, 1991