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Lysosomal Storage Diseases, Nervous System

Clinical trial condition

Lysosomal Storage Diseases, Nervous System

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

513 public trials 105 recruiting studies

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Lysosomal Storage Diseases, Nervous System trial results

Completed

Gene Therapy for Tay-Sachs Disease

NCT01869270

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Data Management and Coordinating Center (DMCC), Univ. of South Florida Tampa, Florida, United States

University of Minnesota Minneapolis, Minnesota, United States

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Completed

Comparison of BMD Measurement by DEXA to BeamMed Speed-of-Sound Measurement at Forearm in Patients With Gaucher Disease

NCT02067247

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations: Shaare Zedek Medical Center Jerusalem, Israel

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Completed

Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease

NCT00025896

Conditions: Acid Maltase Deficiency Disease, Brain Diseases, Brain Diseases, Metabolic, +15 more
Locations: Duke University Medical Center Durham, North Carolina, United States

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Completed

Ophthalmic Findings During 10-year Enzyme Substitution of Danish Fabry Patients.

NCT01997489

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Ulla Feldt-Rasmussen Copenhagen, Capital Region, Denmark

National University Hospital, Department of Medical Endocrinology Copenhagen, Denmark

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Completed

A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta

NCT01650779

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Coral Springs, Florida, United States

Decatur, Georgia, United States

Baltimore, Maryland, United States

Grand Rapids, Michigan, United States

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Completed

A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers

NCT02082327

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: PRA International Groningen, Netherlands

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Completed

Pulmonary Involvement in Patients With Fabry Disease

NCT01632111

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: University Hospital Zurich, Division of Internal Medicine Zurich, Switzerland

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Completed

Cellular and Molecular Mechanisms Governing Bone Marrow Stem Cells in Gaucher Disease

NCT01439607

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations: Bertrand Arnulf Paris, France

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Completed

An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease

NCT00701129

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
Locations: Kosair Children's Hospital Louisville, Kentucky, United States

Duke University Medical Center Durham, North Carolina, United States

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Completed

Safety and Pharmacokinetics of Oral PRX-112 in Gaucher Disease Patients

NCT01747980

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations: Rambam Medical Center Haifa, Israel

Shaare Zedek Medical Center Jerusalem, Israel

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Lysosomal Storage Diseases, Nervous System

Lysosomal Storage Diseases, Nervous System trial results

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