Sandhoff Disease - 14 Studies Found
Completed |
: Stem Cell Transplant for Inborn Errors of Metabolism :
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Completed |
: Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis : Gangliosidoses GM2 : 2007-01-04 : Drug: miglustat Target dose of 320 mg/m^2/day (divided in 3 doses) will be based on the Body Surface Are |
Completed |
: Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders :
: 2012-06-20 :
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Terminated |
: ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases :
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