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Review
. 1999 Nov;6(6):377-82.
doi: 10.1097/00062752-199911000-00004.

Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome

W Krivit  1 , P Aubourg, E Shapiro, C Peters
Affiliations
Review

Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome

W Krivit et al. Curr Opin Hematol. 1999 Nov.

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.

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