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Lysosomal Storage Diseases, Nervous System

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Lysosomal Storage Diseases, Nervous System

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

520 public trials 102 recruiting studies

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Lysosomal Storage Diseases, Nervous System trial results

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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Completed

Paricalcitol in Fabry Disease

NCT02090608

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +29 more
Locations: federico II university, department of nephrology Naples, Italy

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Completed

High Dose or High Dose Frequency Study of Alglucosidase Alfa

NCT00483379

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations: Birmingham, Alabama, United States

Stanford, California, United States

Washington D.C., District of Columbia, United States

Chicago, Illinois, United States

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Completed

Late-Onset Treatment Study Extension Protocol

NCT00455195

Conditions: Acid Maltase Deficiency (AMD), Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations: Anchorage, Alaska, United States

Phoenix, Arizona, United States

Beverly Hills, California, United States

San Diego, California, United States

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Completed

Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®

NCT00731081

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations: Pitie-Salpetriere Hospital Paris, France

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Lysosomal Storage Diseases, Nervous System

Lysosomal Storage Diseases, Nervous System trial results

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