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Lysosomal Storage Diseases

Clinical trial condition

Lysosomal Storage Diseases

Explore clinical trials studying Lysosomal Storage Diseases. Study availability and eligibility vary by location and protocol.

710 public trials 130 recruiting studies

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Lysosomal Storage Diseases trial results

Completed

The Natural History of Infantile Globoid Cell Leukodystrophy

NCT00983879

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
Locations: Children's Hospital of Pittsburgh, 4401 One Children's Hospital Drive,4401 Penn Avenue Pittsburgh, Pennsylvania, United States

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Completed

Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1

NCT02124083

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +21 more
Locations: National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda, Maryland, United States

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Completed

Fabry Screening Study

NCT01019629

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Baylor Institute of Metabolic Disease Dallas, Texas, United States

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Completed

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

NCT01043640

Conditions: Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +52 more
Locations: Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States

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Completed

Safety and Effect of L. Reuteri on Biomarkers of Inflammation in Healthy Infants With Colic

NCT01849991

Conditions: Behavior, Carbohydrate Metabolism, Inborn Errors, Colic, +16 more
Locations: University of Texas Health Science Center at Houston Houston, Texas, United States

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Completed

Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis

NCT02067650

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +11 more
Locations: Children's Hospital of Eastern Ontario Ottawa, Ontario, Canada

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Completed

Stem Cell Transplant for Inborn Errors of Metabolism

NCT00176904

Conditions: Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +55 more
Locations: Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States

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Completed

Stem Cell Transplantation for Hurler

NCT00176917

Conditions: Bone Diseases, Bone Diseases, Metabolic, Brain Diseases, +23 more
Locations: Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States

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Completed

Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III

NCT03161171

Conditions: Behavior Disorders, Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +24 more
Locations: University of Heidelberg Heidelberg, Germany

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Completed

Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry Disease

NCT02995993

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Ruhruniversität Bochum, Klinik für Kinder- und Jugendmedizin im St. Josef-Hospital im Katholischen Klinikum Bochum Bochum, Germany

Universitätsmedizin Mainz, Zentrum für Kinder- und Jugendmedizin Mainz, Germany

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