Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome
W Krivit, P Aubourg, E Shapiro… - Current opinion in …, 1999 - journals.lww.com
W Krivit, P Aubourg, E Shapiro, C Peters
Current opinion in hematology, 1999•journals.lww.comBone 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.
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.
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.
Lippincott Williams & Wilkins