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Lipidoses

Clinical trial condition

Lipidoses

Explore clinical trials studying Lipidoses. Study availability and eligibility vary by location and protocol.

448 public trials 93 recruiting studies

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Lipidoses trial results

Completed

Baby Detect : Genomic Newborn Screening

NCT05687474

Conditions: 3-Hydroxy 3-Methyl Glutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency, +368 more
Locations: CRMN, Hôpital La Citadelle Liège, Wallonia, Belgium

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Completed

A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease

NCT05529992

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations: Chinese PLA General Hospital Beijing, Beijing Municipality, China

Beijing Children's Hospital, Capital Medical University Beijing, Beijing Municipality, China

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences Beijing, Beijing Municipality, China

Lanzhou University Second Hospital Lanzhou, Gansu, China

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Completed

The Natural History of Metachromatic Leukodystrophy Study (HOME Study)

NCT04628364

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +23 more
Locations: National Organization for Rare Disorders Danbury, Connecticut, United States

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Completed

Safety and Efficacy of Miglustat in Chinese NPC Patients

NCT03910621

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Peking University first Hospital Beijing, China

Xin Hua Hospital, Shanghai Jiao Tong University Shanghai, China

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Completed

Autoimmune and Inflammatory Response Biomarkers in Fabry Disease

NCT06007768

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +22 more
Locations: Hospital Universitario Ramón y Cajal Madrid, Spain

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Completed

A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

NCT02678689

Conditions: Batten Disease, CLN2 Disease, CLN2 Disorder, +14 more
Locations: Nationwide Children's Hospital Columbus, Ohio, United States

Universitaetsklinikum Hamburg-Eppendorf Hamburg, Germany

Children's Hospital Bambino Gesù,IRCCS Rome, Piazza, Italy

Great Ormond Street Childrens Hospital London, United Kingdom

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Completed

A Co-designed Physical Activity Intervention in Fabry Disease

NCT06257901

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Brunel University London London, United Kingdom

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Completed

Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France

NCT05359276

Conditions: Acid Sphingomyelinase Deficiency (ASMD), Brain Diseases, Brain Diseases, Metabolic, +19 more
Locations: Investigational site in France France

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Completed

Miglustat in Niemann-Pick Type C Disease

NCT00517153

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations: Not specified

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Completed

Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease

NCT00319046

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations: Not specified

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