498 studies found for:    "Lysosomal Storage Diseases" OR "Schindler disease"
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Rank Status Study
1 Active, not recruiting Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases
Conditions: Lysosomal Storage Diseases;   Inborn Errors of Metabolism
Intervention: Biological: ALD-601
2 Enrolling by invitation Genetic Studies of Lysosomal Storage Disorders
Conditions: Gaucher's Disease;   Lysosomal Storage Disease
Intervention:
3 Recruiting Investigating Lysosomal Storage Diseases in Minority Groups
Conditions: Lysosomal Storage Disorders;   Gaucher Disease;   Fabry Disease;   Pompe Disease;   Niemann-Pick Disease
Intervention:
4 Recruiting Molecular and Cellular Mechanisms of Lysosomal Storage Diseases
Condition: Lysosomal Storage Disorders
Intervention:
5 Recruiting Biomarker for Krabbe Disease
Conditions: Lysosomal Storage Diseases;   Krabbe Disease
Intervention:
6 Recruiting Biomarker for Pompe Disease
Conditions: Lysosomal Storage Diseases;   Pompe Disease
Intervention:
7 Recruiting Longitudinal Studies of the Glycoproteinoses
Conditions: Aspartylglucosaminuria;   Fucosidosis;   Galactosialidosis;   Alpha Mannosidosis;   Beta Mannosidosis;   Mucolipidosis II;   Mucolipidosis III;   Schindler Disease;   Sialidosis
Intervention:
8 Recruiting Biomarker for Patients With Fabry Disease
Conditions: Fabry Disease;   Anderson-Fabry Disease;   Lysosomal Storage Diseases;   Alpha-Galactosidase A Deficiency
Intervention:
9 Recruiting LYSO-PROVE - Determine the Prognostic Value of Lyso-Gb1 for Monitoring the Progress of Gaucher Disease
Conditions: Lysosomal Storage Diseases;   Gaucher Disease;   Sphingolipidoses
Intervention:
10 Not yet recruiting Diet and Exercise in Pompe Disease
Conditions: Pompe Disease;   Glycogen Storage Disease Type II;   Lysosomal Storage Diseases
Intervention: Other: Diet and Exercise
11 Terminated Natural History Study of Children With Metachromatic Leukodystrophy
Conditions: Lipid Metabolism Disorders;   Metachromatic Leukodystrophy (MLD);   Nervous System Diseases;   Brain Diseases;   Central Nervous System Diseases;   Demyelinating Diseases;   Metabolism, Inborn Errors;   Genetic Diseases, Inborn;   Sphingolipidoses;   Hereditary Central Nervous System Demyelinating Diseases;   Metabolic Inborn Brain Diseases;   Lysosomal Storage Diseases;   Metabolic Diseases;   Sulfatidosis
Intervention:
12 Recruiting Biomarker for Gaucher Disease
Conditions: Lysosomal Storage Diseases;   Gaucher Disease;   Sphingolipidoses
Intervention:
13 Recruiting Biomarker for Morquio Disease
Conditions: Lysosomal Storage Diseases;   Morquio Disease
Intervention:
14 Recruiting Biomarker for Hunter Disease
Conditions: Lysosomal Storage Diseases;   Hunter Disease;   Mucopolysaccharidoses
Intervention:
15 Recruiting Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children
Conditions: Mucopolysaccharidosis Type I (MPS I);   Mucopolysaccharidosis Type II (MPS II);   Mucopolysaccharidosis Type III (MPS III);   Mucopolysaccharidosis Type VI (MPS VI);   Krabbe Disease
Intervention:
16 Recruiting Biomarker for Metachromatic Leukodystrophy Disease
Conditions: Leukodystrophy, Metachromatic;   Hereditary Central Nervous System Demyelinating Diseases;   Brain Diseases, Metabolic, Inborn;   Sphingolipidoses;   Lysosomal Storage Diseases, Nervous System
Intervention:
17 Completed Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
Conditions: Lysosomal Storage Disease;   Peroxisomal Disorder
Interventions: Drug: Campath-1H;   Drug: Clofarabine;   Drug: Melphalan;   Radiation: Total Body Irradiation with Marrow Boosting;   Biological: Hematopoietic stem cell transplantation;   Drug: Cyclosporine A;   Drug: Mycophenolate mofetil
18 Completed A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
Conditions: Cognitive Decline;   Mucopolysaccharidosis I;   Hurler-Scheie Syndrome;   Scheie Syndrome;   Lysosomal Storage Disease
Intervention: Drug: laronidase
19 Terminated Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I
Conditions: Spinal Cord Compression;   Mucopolysaccharidosis I;   Hurler-Scheie Syndrome;   Scheie Syndrome;   Lysosomal Storage Disease
Intervention: Drug: laronidase
20 Completed
Has Results
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
Conditions: Non Malignant Disorders;   Immunodeficiencies;   Congenital Marrow Failures;   Hemoglobinopathies;   Inborn Errors of Metabolism;   Sickle Cell;   Thalassemia;   Lysosomal Storage Disease
Interventions: Biological: Unrelated Umbilical Cord Blood Transplant;   Drug: Reduced Intensity Conditioning

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Indicates status has not been verified in more than two years