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Found 12 studies with search of: Tay-Sachs Disease
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Rank Status Study
1 Recruiting A Natural History Study of Hexosaminidase Deficiency in Infantile Tay-Sachs Disease
Condition: Tay Sachs Disease
Intervention:
2 Suspended A Phase I Study of Pyrimethamine in Patients With GM-2 Gangliosidosis
Conditions: G(M2) Ganglioside;   Tay-Sachs Disease Ganglioside;   Sandhoff Disease Ganglioside
Intervention: Drug: Pyrimethamine
3 Active, not recruiting Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
Condition: Gangliosidoses GM2
Intervention: Drug: miglustat
4 Completed Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses
Conditions: GM2 Gangliosidoses;   Tay-Sachs;   Sandhoff Disease
Intervention: Drug: Zavesca (Miglustat)
5 Recruiting HSCT for High Risk Inherited Inborn Errors
Conditions: Adrenoleukodystrophy;   Metachromatic Leukodystrophy;   Globoid Cell Leukodystrophy;   Tay Sachs Disease;   Sandhoffs Disease;   I-Cell Disease;   Sanfillipo;   GM1 Gangliosidosis
Interventions: Drug: Campath;   Drug: Clofarabine;   Procedure: Total body Irradiation;   Drug: Melphalan
6 Completed Diagnostic and Screening Study of Genetic Disorders
Conditions: Tay-Sachs Disease;   Porphyria, Erythropoietic;   Leukodystrophy, Globoid Cell;   Metabolism, Inborn Errors
Intervention:
7 Active, not recruiting Stem Cell Transplant for Inborn Errors of Metabolism
Conditions: Adrenoleukodystrophy;   Metachromatic Leukodystrophy;   Globoid Cell Leukodystrophy;   Gaucher's Disease;   Fucosidosis;   Wolman Disease;   Niemann-Pick Disease;   Batten Disease;   GM1 Gangliosidosis;   Tay Sachs Disease;   Sandhoff Disease
Interventions: Procedure: Stem Cell Transplant;   Drug: Busulfan, Cyclophosphamide, ATG
8 Suspended ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Conditions: Inherited Metabolic Diseases;   Lysosomal Storage Disorders;   Peroxisomal Storage Diseases;   Inborn Errors of Metabolism;   Mucopolysaccharidosis
Intervention: Biological: ALD-101
9 Recruiting Nervous System Degeneration in Glycosphingolipid Storage Disorders
Conditions: Gangliosidoses;   Gaucher Disease
Intervention:
10 Recruiting Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases
Conditions: Lysosomal Storage Diseases;   Inborn Errors of Metabolism
Intervention: Biological: ALD-601
11 Recruiting Single Gene Preimplantation Genetic Diagnosis (PGD)
Condition: Any Single Gene Disorder (Cystic Fibrosis, Tay-Sachs)
Intervention: Other: preimplantation diagnosis
12 Recruiting Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells
Conditions: MDS;   Anemia, Aplastic;   Inborn Errors of Metabolism;   Congenital Marrow Failure;   Congenital Immunodeficiency Syndrome
Intervention: Biological: ALDHbr Umbilical Cord Blood Cells

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