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Found 174 studies with search of: "Lipid Storage Diseases"
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Rank Status Study
1 Completed
Has Results
Lipid Infusion in Dialysis Patients With Endotoxemia
Conditions: Fatigue;   End Stage Renal Disease (ESRD)
Interventions: Drug: Lipidose;   Drug: Placebo
2 Enrolling by invitation The Effect of Fibrate Therapy in Two Patients With Neutral Lipid Storage Disease With Myopathy (NLSDM)
Condition: Neutral Lipid Storage Disease
Intervention: Drug: Fibrate treatment
3 Recruiting Study of Inborn Errors of Cholesterol Synthesis and Related Disorders
Condition: Lipoidosis
Intervention:
4 Completed SCH-58235 (Ezetimibe) to Treat Homozygous Sitosterolemia
Condition: Lipoidosis
Intervention: Drug: SCH-58235
5 Completed A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.
Conditions: Gaucher Disease Type I;   Cerebroside Lipidosis Syndrome;   Clucocerebrosidase Deficiency Disease;   Glucosylceramide Beta-Glucosidase Deficiency Disease;   Gaucher Disease, Non-Neuronopathic Form
Intervention: Drug: Cerezyme (imiglucerase for injection)
6 Recruiting International Collaborative Gaucher Group (ICGG) Gaucher Registry
Conditions: Gaucher Disease;   Cerebroside Lipidosis Syndrome;   Glucocerebrosidase Deficiency Disease;   Glucosylceramide Beta-Glucosidase Deficiency Disease
Intervention:
7 Active, not recruiting A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients
Conditions: Gaucher Disease, Type 1;   Cerebroside Lipidosis Syndrome;   Glucocerebrosidase Deficiency Disease;   Glucosylceramide Beta-Glucosidase Deficiency Disease;   Gaucher Disease, Non-Neuronopathic Form
Intervention: Drug: eliglustat tartrate
8 Completed
Has Results
Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease
Conditions: Gaucher Disease, Type 1;   Cerebroside Lipidosis Syndrome;   Glucocerebrosidase Deficiency Disease;   Glucosylceramide Beta-Glucosidase Deficiency Disease;   Gaucher Disease, Non-Neuronopathic Form
Intervention: Drug: Cerezyme
9 Recruiting Biomarker for Niemann Pick Type C Disease
Conditions: Niemann-Pick Disease;   Niemann-Pick Disease, Type C
Intervention:
10 Recruiting Biomarker for Gaucher Diasease
Conditions: Lysosomal Storage Diseases;   Gaucher Disease;   Sphingolipidoses
Intervention:
11 Recruiting Biomarker for Krabbe Disease
Conditions: Lysosomal Storage Diseases;   Krabbe Disease
Intervention:
12 Completed Registry of Fabry Disease - A Multicenter Observational Study
Condition: Fabry Disease
Intervention:
13 Unknown  Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
Conditions: I Cell Disease;   Fucosidosis;   Globoid Cell Leukodystrophy;   Adrenoleukodystrophy;   Mannosidosis;   Niemann-Pick Disease;   Pulmonary Complications;   Mucopolysaccharidosis I;   Mucopolysaccharidosis VI;   Metachromatic Leukodystrophy;   Gaucher's Disease;   Wolman Disease
Intervention:
14 Completed A Study of AT1001 in Patients With Fabry Disease
Condition: Fabry Disease
Intervention: Drug: AT1001 (migalastat hydrochloride)
15 Active, not recruiting Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
Condition: Fabry Disease
Interventions: Drug: migalastat hydrochloride;   Drug: Placebo
16 Active, not recruiting A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)
Condition: Gaucher Disease, Type 1
Interventions: Drug: eliglustat tartrate;   Drug: Placebo
17 Completed
Has Results
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, Antithymocyte Globulin
18 Recruiting Canadian Fabry Disease Initiative (CFDI) Enzyme Replacement Therapy (ERT) Study
Condition: Fabry Disease
Interventions: Drug: agalsidase alfa (Replagal®) and agalsidase beta (Fabrazyme®);   Drug: Enzyme Replacement Therapy (ERT)
19 Enrolling by invitation Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)
Condition: Gaucher Disease
Intervention: Drug: Imiglucerase
20 Withdrawn An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD)
Condition: Leukodystrophy, Globoid Cell
Intervention:

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