Deficiency Diseases - 55 Studies Found
Completed |
: IMM 0212: Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell Immunodeficiency :
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Completed |
: Construction of a Health-related Quality of Life (HR-QOL) Questionnaire for Patients With Primary Antibody Deficiency Disease : Immune Deficiency, Antibody : 2015-08-31 : Other: questionnaire survey The HR-QOL PIDD survey will be administered to the patient by the site PI or |
Completed |
: GTA-Glyceryltriacetate for Canavan Disease :
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Recruiting |
: International Collaborative Gaucher Group (ICGG) Gaucher Registry :
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Completed |
: A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease. :
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Completed |
: Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease :
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Completed |
: A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients :
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Active, not recruiting |
: Growth and Development Study of Alglucosidase Alfa. :
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Withdrawn |
: Lithium and Acetate for Canavan Disease :
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Approved for marketing |
: Alglucosidase Alfa Temporary Access Program :
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