Clinical Trials on Metabolic Inborn Brain Diseases
Total 87528 results
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Duke UniversityCompleted
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Duke UniversityGenzyme, a Sanofi CompanyCompleted
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Duke UniversityCompletedPompe DiseaseUnited States
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Duke UniversityCompleted
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Dwight Koeberl, M.D., Ph.D.Completed
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University of California, IrvineGenzyme, a Sanofi CompanyCompletedPompe DiseaseUnited States
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Amicus TherapeuticsTerminated
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University of FloridaTerminated
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O & O Alpan LLCCompleted
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Genzyme, a Sanofi CompanyCompleted
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type IIUnited States, France, Netherlands
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National Taiwan University HospitalUnknown
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Children's Healthcare of AtlantaTerminatedCongenital Disorders
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Ludwig-Maximilians - University of MunichCompletedPhenylketonuria | Nutritional DeficienciesGermany
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Genzyme, a Sanofi CompanyActive, not recruitingGaucher Disease Type 1 | Gaucher Disease Type 3Germany, United States, Japan, United Kingdom
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KemPharm Denmark A/STerminatedGaucher Disease, Type 1 | Gaucher Disease, Type 3India
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Baylor Research InstituteTexas Scottish Rite Hospital for ChildrenWithdrawnGaucher Disease Type 1 | Gaucher Disease Type 3United States
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Children's Hospital of PhiladelphiaEunice Kennedy Shriver National Institute of Child Health and Human Development...WithdrawnUrea Cycle Disorders, Inborn | Inborn Errors of Metabolism | Propionic Acidemia | Methylmalonic Acidemia | Carbamyl Phosphate Synthetase Deficiency
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Horizon Pharma Ireland, Ltd., Dublin IrelandUcyclyd Pharma, Inc.CompletedHepatic Encephalopathy | Urea Cycle DisordersUkraine
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University of AarhusRecruiting
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SanofiRecruitingGaucher's Disease Type IIIUnited States, Germany, Hungary, France, China, Japan, Canada, Argentina, Italy, Turkey
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GeneCradle IncRecruitingPompe Disease (Late-onset)China
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GeneCradle IncRecruitingPompe Disease Infantile-OnsetChina
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Seventh Medical Center of PLA General HospitalGeneCradle Therapeutics, IncRecruiting
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Duke UniversityCompleted
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Fundació Institut de Recerca de l'Hospital de la...Genzyme, a Sanofi CompanyUnknownPompe Disease | Late Onset Pompe DiseaseSpain
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Genzyme, a Sanofi CompanyActive, not recruitingTay-Sachs Disease | Sandhoff DiseaseUnited States, Argentina, Czechia, Portugal, Russian Federation, Turkey, United Kingdom, Brazil, France, Germany, Italy, Japan, Spain
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Azafaros A.G.Active, not recruitingGM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Brazil, France, Germany, Italy, United Kingdom
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Astellas Gene TherapiesRecruitingPompe Disease (Late-onset)United States, Spain, France, Taiwan, Italy
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Amicus TherapeuticsCompletedPompe Disease (Late-onset)United States, Australia, Korea, Republic of, Japan, Taiwan, Belgium, Slovenia, New Zealand, United Kingdom, France, Argentina, Canada, Spain, Bosnia and Herzegovina, Germany, Sweden, Hungary, Austria, Bulgaria, Denmark, Greece, I... and more
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Duke UniversityCompleted
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Amicus TherapeuticsTerminatedLate-onset Pompe DiseaseUnited States, Canada, Australia, Belgium
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BioMarin PharmaceuticalTerminatedLate-onset Pompe DiseaseUnited States, United Kingdom, Germany, France, Italy, Portugal, Belgium, Netherlands
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGaucher Disease Type 1United States
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Children's National Research InstituteActelionCompletedSandhoff Disease | GM2 Gangliosidoses | Tay-SachsUnited States
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Children's Hospital Medical Center, CincinnatiCompletedOsteopenia | Gaucher's DiseaseIsrael, United States
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Genzyme, a Sanofi CompanyCompletedPompe DiseaseBelgium, United States, Czechia, Germany, Italy, Taiwan
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University of FloridaAmicus TherapeuticsTerminatedPompe Disease | Hypersensitivity Reaction
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Aeglea BiotherapeuticsTerminatedHomocystinuria Due to Cystathionine Beta-Synthase DeficiencyUnited States, United Kingdom, Australia
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Promethera TherapeuticsCompletedUrea Cycle Disorders | Crigler Najjar SyndromeBelgium
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Amicus TherapeuticsRecruitingGlycogen Storage Disease Type II Infantile OnsetUnited States, Taiwan, France, Germany, Italy, United Kingdom
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Glycogenosis 2United States
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Hacettepe UniversityCompletedCaregiver Burden | Life Stress | Psychological Well-BeingTurkey
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Emory UniversityRecruiting
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Vitaflo International, LtdCompletedAlkaptonuria | Tyrosinemias | Tyrosinemia, Type I | Tyrosinemia, Type II | Tyrosinemia, Type IIIUnited Kingdom
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Horizon Pharma USA, Inc.CompletedInherited Mitochondrial Disease, Including Leigh SyndromeUnited States
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Forge Biologics, IncNot yet recruitingKrabbe DiseaseUnited States
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Forge Biologics, IncActive, not recruiting
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University of PittsburghNational Institute of Neurological Disorders and Stroke (NINDS)Active, not recruiting