Sandhoff disease

Search Trials
Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
Status:
Completed
Last Changed:
Dec 5, 2017
First Changed:
Jun 22, 2012
Disease(s):
Lysosomal Storage Disease
Intervention(s):
Campath-1HClofarabineMelphalanTotal Body Irradiation with Marrow BoostingHematopoietic stem cell transplantationCyclosporine AMycophenolate mofetil
Synergistic Enteral Regimen for Treatment of the Gangliosidoses
Status:
Recruiting
Last Changed:
May 29, 2019
First Changed:
Jan 8, 2014
Disease(s):
GM1 Gangliosidoses
Intervention(s):
miglustatKetogenic Diet
Longitudinal Study of Neurodegenerative Disorders
Status:
Recruiting
Last Changed:
Dec 5, 2018
First Changed:
Nov 6, 2017
Disease(s):
MLD
Intervention(s):
Palliative CareHematopoetic Stem Cell Transplantation
Stem Cell Transplant for Inborn Errors of Metabolism
Status:
Completed
Last Changed:
Dec 28, 2017
First Changed:
Sep 15, 2005
Disease(s):
Adrenoleukodystrophy
Intervention(s):
Stem Cell TransplantBusulfan, Cyclophosphamide, Antithymocyte Globulin

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