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Found 227 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 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
6 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
7 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)
8 Recruiting International Collaborative Gaucher Group (ICGG) Gaucher Registry
Conditions: Gaucher Disease;   Cerebroside Lipidosis Syndrome;   Glucocerebrosidase Deficiency Disease;   Glucosylceramide Beta-Glucosidase Deficiency Disease
Intervention:
9 Recruiting Biomarker for Krabbe Disease
Conditions: Lysosomal Storage Diseases;   Krabbe Disease
Intervention:
10 Recruiting Biomarker for Gaucher Disease
Conditions: Lysosomal Storage Diseases;   Gaucher Disease;   Sphingolipidoses
Intervention:
11 Recruiting Biomarker for Niemann Pick Type C Disease
Conditions: Niemann-Pick Disease;   Niemann-Pick Disease, Type C
Intervention:
12 Completed Registry of Fabry Disease - A Multicenter Observational Study
Condition: Fabry Disease
Intervention:
13 Completed An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease
Condition: Fabry Disease
Interventions: Drug: Replagal agalsidase alfa;   Drug: Replagal
14 Completed An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease
Condition: Fabry Disease
Intervention: Drug: Replagal
15 Enrolling by invitation Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 in Adult Subjects With Lysosomal Acid Lipase Deficiency
Conditions: Cholesterol Ester Storage Disease(CESD);   Lysosomal Acid Lipase Deficiency
Intervention: Drug: SBC-102
16 Completed PEG-Glucocerebrosidase for the Treatment of Gaucher Disease
Condition: Gaucher's Disease
Intervention: Drug: Lysodase
17 Recruiting An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype
Conditions: Cholesterol Ester Storage Disease(CESD);   Lysosomal Acid Lipase Deficiency
Intervention:
18 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
19 Completed Safety, Tolerability and Pharmacokinetics of SBC-102 in Adult Patients With Lysosomal Acid Lipase Deficiency
Conditions: Cholesterol Ester Storage Disease(CESD);   Lysosomal Acid Lipase Deficiency
Intervention: Drug: SBC-102
20 Completed OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease
Condition: Gaucher Disease
Intervention: Drug: OGT 918

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