Clinical Trials on Brain Diseases, Metabolic, Inborn
Total 86698 results
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Emory UniversityPatient-Centered Outcomes Research InstituteCompleted
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Cycle Pharmaceuticals Ltd.ParexelCompletedHereditary Tyrosinemia, Type ISouth Africa
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University of ZurichCompletedPhenylketonuria (PKU) and HyperphenylalaninemiaSwitzerland
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Swedish Orphan BiovitrumCompletedHereditary Tyrosinemia, Type IGermany, United Kingdom, France
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Orphanetics Pharma Entwicklungs GmbHTerminatedPhenylalanine Hydroxylase Deficiencies
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National Center for Research Resources (NCRR)University of TexasCompleted
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University Hospital, ToursCompletedAdult Phenylketonuria Non Treated PatientsFrance
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Swedish Orphan BiovitrumCompletedHereditary Tyrosinemia, Type IBelgium, Denmark, France, Germany, Austria, Czechia, Finland, Hungary, Ireland, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, United Kingdom
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Cycle Pharmaceuticals Ltd.ParexelCompletedHereditary Tyrosinemia, Type ISouth Africa
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ShireTerminated
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TakedaCompleted
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Genzyme, a Sanofi CompanyCompletedGaucher DiseaseCanada, Russian Federation, Tunisia
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ShireCompleted
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PfizerCompletedGaucher DiseaseUnited Kingdom, Canada, Israel, United States, Australia, Spain
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PfizerCompletedGaucher DiseaseChile, United Kingdom, Canada, Spain, Israel, United States, Australia, South Africa
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PfizerCompletedGaucher DiseaseIsrael, Paraguay, South Africa
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Yasmeen Abd Elbaset HamzaaUnknownGaucher Disease
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Genzyme, a Sanofi CompanyCompletedGaucher DiseaseUnited States
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Genzyme, a Sanofi CompanyCompletedGaucher DiseaseUnited States, Sweden, India, Netherlands, Austria, Japan, China, Serbia, France, Croatia, Australia, Portugal, Canada, Brazil, Greece, Romania, Russian Federation
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ISU Abxis Co., Ltd.Completed
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Ultragenyx Pharmaceutical IncCompletedOrnithine Transcarbamylase DeficiencyUnited 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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Emma Marie Caroline SlackUniversity Children's HospitalCompletedInborn Errors of Metabolism | Propionic Aciduria | Methylmalonic Aciduria | Urea Cycle DisorderSwitzerland
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Aeglea BiotherapeuticsTerminatedArginase I Deficiency | HyperargininemiaCanada
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Italy, Canada, France, United Kingdom, Austria, Germany
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Children's National Research InstitutePatient-Centered Outcomes Research Institute; Eunice Kennedy Shriver National... and other collaboratorsCompleted
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Vitaflo International, LtdActive, not recruiting
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Cycle Pharmaceuticals Ltd.ParexelCompletedHereditary Tyrosinemia, Type ISouth Africa
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Swedish Orphan BiovitrumCompletedEfficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1 (HT-1)Hereditary Tyrosinemia, Type IBelgium, Denmark, France, Germany, Sweden
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CENTOGENE GmbH RostockWithdrawnMetabolic Disorders | Tyrosinosis | Hepatorenal Tyrosinemia | Fumarylacetoacetase Deficiency | Fah DeficiencyGermany, Sri Lanka, India
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Istanbul University - Cerrahpasa (IUC)CompletedUrea Cycle Disorder | Lysinuric Protein IntoleranceTurkey
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BioMarin PharmaceuticalCompletedTetrahydrobiopterin Deficiencies | Hyperphenylalaninemia, Non-PhenylketonuricGermany, United States
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Eunice Kennedy Shriver National Institute of Child...Completed
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SanofiCompletedGaucher's DiseaseUnited States
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ShireCompletedGaucher DiseaseUnited States, Spain, United Kingdom, Israel
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ShireCompletedGaucher DiseaseIsrael, Romania, Serbia
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ShireCompletedGaucher Disease, Type 3Egypt, India, Tunisia
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ISU Abxis Co., Ltd.CompletedGaucher Disease, Type 1
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Genzyme, a Sanofi CompanyCompletedGaucher Disease, Type 1United States, United Kingdom, Bulgaria, Canada, Colombia, India, Israel, Lebanon, Mexico, Russian Federation, Serbia, Tunisia
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Genzyme, a Sanofi CompanyCompletedGaucher Disease, Type 1United States, France, Germany, Italy, Brazil, Australia, Argentina, Canada, Egypt, Russian Federation, Spain, United Kingdom
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Genzyme, a Sanofi CompanyWithdrawnGaucher Disease, Type 1United States, India
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ISU Abxis Co., Ltd.Completed
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Amicus TherapeuticsTerminatedType 1 Gaucher DiseaseUnited States, United Kingdom
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National Institute of Neurological Disorders and...Completed
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University of GlasgowCompletedPhenylketonuria (PKU)United Kingdom
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University of WashingtonCompleted
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National Center for Research Resources (NCRR)University of MichiganCompletedTyrosinemia, Type I