Clinical Trials on Brain Diseases, Metabolic in United States
Total 147767 results
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Emory UniversityNational PKU AllianceCompleted
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Emory UniversityPatient-Centered Outcomes Research InstituteCompleted
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BioMarin PharmaceuticalRecruitingPhenylketonuria (PKU)United States
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University of Southern CaliforniaBioMarin PharmaceuticalCompleted
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SanofiCompletedGaucher's DiseaseUnited States
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National Institute of Mental Health (NIMH)Completed
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National Institute of Neurological Disorders and...CompletedGaucher DiseaseUnited States
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Columbia UniversityWithdrawnGlycogen Storage Disease Type IIUnited States
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Genzyme, a Sanofi CompanyCompletedGaucher DiseaseUnited States
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Nutricia ResearchCompletedInborn Errors of Metabolism | Urea Cycle DisorderUnited States
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Baylor College of MedicineCompletedUrea Cycle Disorders, InbornUnited States
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Dr. Linda RandolphBioMarin PharmaceuticalTerminated
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Homology Medicines, IncTerminatedPhenylketonuria | Phenylketonurias | PAH DeficiencyUnited States
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National Institute of Neurological Disorders and...CompletedGaucher's DiseaseUnited States
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University of South FloridaNational Institutes of Health (NIH)CompletedUrea Cycle DisordersUnited States
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Baylor College of MedicineCompletedCitrullinemiaUnited States
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National Center for Research Resources (NCRR)National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); University...Completed
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Exsar CorporationSuspendedType I Gaucher DiseaseUnited States
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SanofiRecruiting
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Lysosomal and Rare Disorders Research and Treatment...UnknownGaucher Disease Type 1United States
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Brendan LeeEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompletedAmino Acid Metabolism, Inborn Errors | Urea Cycle Disorders | Argininosuccinic AciduriaUnited States
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National Center for Research Resources (NCRR)University of PittsburghUnknownGaucher's DiseaseUnited States
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LYSOGENECasimir, LLC; Cure GM1 FoundationCompleted
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Duke UniversityNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)CompletedGlycogen Storage Disease Type IIUnited States
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Applied Therapeutics, Inc.Active, not recruitingClassic GalactosemiaUnited States
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Children's Hospital of PhiladelphiaActive, not recruitingMaple Syrup Urine DiseaseUnited States
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Genzyme, a Sanofi CompanyWithdrawnGaucher Disease, Type 1United States, India
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BioMarin PharmaceuticalActive, not recruiting
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Homology Medicines, IncEnrolling by invitationPhenylketonuria | PAH DeficiencyUnited States
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Children's National Research InstituteBoston Children's HospitalRecruitingUrea Cycle DisordersUnited States
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University of Missouri-ColumbiaCompleted
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Massachusetts General HospitalActive, not recruiting
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ModernaTX, Inc.RecruitingPhenylketonuriaUnited States, Australia
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Lysosomal and Rare Disorders Research and Treatment...RecruitingBone Diseases | Gaucher DiseaseUnited States
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National Institute of Neurological Disorders and...CompletedGaucher DiseaseUnited States, United Kingdom
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University of FloridaAmicus TherapeuticsCompletedLysosomal Storage Diseases | Glycogen Storage Disease Type II | Pompe DiseaseUnited States
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University of FloridaNational Heart, Lung, and Blood Institute (NHLBI)CompletedPompe DiseaseUnited States
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University of FloridaCompletedPompe DiseaseUnited States
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Horizon Pharma Ireland, Ltd., Dublin IrelandCompletedUrea Cycle DisordersUnited States
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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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Baylor Research InstituteTexas Scottish Rite Hospital for ChildrenWithdrawnGaucher Disease Type 1 | Gaucher Disease Type 3United States
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Horizon Therapeutics, LLCTerminatedUrea Cycle DisorderUnited States
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University of FloridaEunice Kennedy Shriver National Institute of Child Health and Human Development...Completed
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University of FloridaLacerta TherapeuticsCompletedPompe DiseaseUnited States
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University of FloridaBioMarin PharmaceuticalCompleted
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Ultragenyx Pharmaceutical IncCompletedOrnithine Transcarbamylase DeficiencyUnited States
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LYSOGENETerminatedGM1 GangliosidosisUnited Kingdom, United States, France
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Applied Therapeutics, Inc.CompletedClassic GalactosemiaUnited States
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Travere Therapeutics, Inc.Enrolling by invitation