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Leukodystrophy, Metachromatic clinical trials

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NCT ID: NCT00004378 Completed - Thrombocytopenia Clinical Trials

Stem Cell Transplantation (SCT) for Genetic Diseases

Start date: January 1995
Phase: N/A
Study type: Interventional

OBJECTIVES: I. Ascertain whether stem cell transplantation (SCT) is an effective method by which missing or dysfunctional enzymes can be replaced in patients with various inborn errors of metabolism. II. Determine whether clinical manifestations of the specific disease may be arrested or reversed by this treatment.