- ICH GCP
- US Clinical Trials Registry
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Clinical Trials on Brain Diseases, Metabolic
Total 379953 results
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Canada, United Kingdom, Portugal
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The Hospital for Sick ChildrenBioMarin PharmaceuticalCompletedPhenylketonuria | Mild HyperphenylalaninemiaCanada
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IntraBio IncCompletedGM2 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Germany, Spain, United Kingdom
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Terence FlotteMassachusetts General Hospital; University of Massachusetts, WorcesterActive, not recruitingTay-Sachs Disease | Sandhoff DiseaseUnited States
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National Institute of Neurological Disorders and...CompletedGaucher DiseaseUnited States, United Kingdom
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University of PennsylvaniaPassage Bio, Inc.RecruitingGM1 GangliosidosisUnited States, Turkey, Brazil, Canada, United Kingdom
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Ultragenyx Pharmaceutical IncRecruitingOTC DeficiencyUnited States, Portugal, Australia, Italy, Spain, United Kingdom, Japan, Canada, Germany, France, Argentina, Netherlands, Brazil
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University College, LondonRecruitingOrnithine Transcarbamylase DeficiencyUnited Kingdom
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Arcturus Therapeutics, Inc.Active, not recruitingOrnithine Transcarbamylase DeficiencyUnited States
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Horizon Pharma Ireland, Ltd., Dublin IrelandCompletedUrea Cycle DisordersUnited States
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Horizon Pharma Ireland, Ltd., Dublin IrelandCompletedUrea Cycle DisordersUnited States, Canada
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Centre Hospitalier de CayenneCompleted
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Massachusetts General HospitalActive, not recruiting
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SanofiCompletedGaucher DiseaseUnited States
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Ultragenyx Pharmaceutical IncCompletedOrnithine Transcarbamylase DeficiencyUnited States
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Assiut UniversityNot yet recruiting
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Tehran University of Medical SciencesMashhad University of Medical Sciences; Kashan University of Medical SciencesRecruitingEffects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) (EMTISTD)Supportive Care | GM2 GangliosidosisIran, Islamic Republic of
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SanofiCompletedGaucher Disease | SplenomegalyFrance
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The Hospital for Sick ChildrenActelionCompletedGangliosidoses GM2Canada
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Rambam Health Care CampusUnknown
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Centre for Analytical Biochemistry and Biomedical...University of GiessenRecruiting
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University of FloridaAmicus TherapeuticsCompletedLysosomal Storage Diseases | Glycogen Storage Disease Type II | Pompe DiseaseUnited States
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Horizon Therapeutics, LLCCompletedUrea Cycle DisordersUnited States, Canada
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Horizon Pharma Ireland, Ltd., Dublin IrelandCompletedUrea Cycle DisordersUnited States, Canada
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National Human Genome Research Institute (NHGRI)RecruitingMitochondrial Disorders | Leigh Disease | Oxidative Phosphorylation Deficiencies | Electron Transport Chain Disorders, MitochondrialUnited States
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Lianyungang Oriental HospitalThe First People's Hospital of LianyungangUnknownHyperglycemia | Brain InjuriesChina
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Chang Gung Memorial HospitalRecruitingNon-Phenylketonuric HyperphenylalaninemiaTaiwan
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Travere Therapeutics, Inc.Active, not recruiting
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SynlogicCompletedHomocystinuriaUnited States
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Assistance Publique - Hôpitaux de ParisCompleted
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Amicus TherapeuticsRecruitingPompe DiseaseUnited States
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Duke UniversitySanofiRecruiting
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Duke UniversityGenzyme, a Sanofi CompanyRecruitingPompe DiseaseUnited States
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Genzyme, a Sanofi CompanyCompleted
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Valerion Therapeutics, LLCTerminatedPompe DiseaseUnited States, United Kingdom
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University of FloridaNational Heart, Lung, and Blood Institute (NHLBI)CompletedPompe DiseaseUnited States
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BioMarin PharmaceuticalCompletedPompe DiseaseUnited States, United Kingdom, France, Australia, Germany
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BioMarin PharmaceuticalTerminatedPompe DiseaseUnited States, United Kingdom, Australia, France, Germany, New Zealand
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University of FloridaCompletedPompe DiseaseUnited States
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National Taiwan University HospitalUnknown
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Genzyme, a Sanofi CompanyTerminatedPompe DiseaseUnited States
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Pompe DiseaseUnited States
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type IIUnited States, Taiwan, Israel, United Kingdom, France
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Children's Hospital Medical Center, CincinnatiCompletedOsteopenia | Gaucher's DiseaseIsrael, United States
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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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Horizon Therapeutics, LLCCompletedUrea Cycle DisorderUnited States, Spain, Italy, Switzerland