Clinical Trials on Brain Diseases, Metabolic, Inborn
Total 87196 results
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Canada, United Kingdom, Portugal
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metaX Institut fuer Diatetik GmbHBirmingham Children's HospitalCompletedPhenylketonurias | Hyperphenylalaninaemia | Tetrahydrobiopterin DeficiencyUnited Kingdom
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BioMarin PharmaceuticalActive, not recruitingPhenylketonuria (PKU)United States, Germany
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Homology Medicines, IncEnrolling by invitationPhenylketonuria | PAH DeficiencyUnited States
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BioMarin PharmaceuticalCompletedPhenylketonuria (PKU)United States
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BioMarin PharmaceuticalCompletedPhenylketonuria (PKU)United States
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University of British ColumbiaRare Disease Foundation, Vancouver, CanadaCompleted
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University of GlasgowUnknownPhenylketonuria (PKU)United Kingdom
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University Hospital, ToursInstitut National de la Santé Et de la Recherche Médicale, FranceCompletedPKU | HyperphenylalaninemiaFrance
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Dr. Linda RandolphBioMarin PharmaceuticalTerminated
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University of MinnesotaRecruitingGaucher DiseaseUnited States
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TakedaNot yet recruiting
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Duke UniversityRecruiting
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TakedaCompletedGaucher DiseaseUnited States
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ShireCompletedGaucher DiseaseIndia
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PfizerCompleted
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ShireQuintiles, Inc.Completed
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ISU Abxis Co., Ltd.CompletedGaucher DiseaseAustralia
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ProtalixCompleted
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ISU Abxis Co., Ltd.Withdrawn
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Genzyme, a Sanofi CompanyCompleted
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ProtalixCompleted
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University Hospital, Clermont-FerrandUnknown
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Brigham and Women's HospitalGenzyme, a Sanofi CompanyUnknown
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ProtalixCompleted
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TakedaActive, not recruiting
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CENTOGENE GmbH RostockActive, not recruiting
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Lysosomal and Rare Disorders Research and Treatment...UnknownGaucher DiseaseUnited States
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PfizerCompletedGaucher DiseaseIsrael, South Africa, Paraguay
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PfizerCompletedGaucher DiseaseUnited States, Chile, United Kingdom, Israel, Canada, Spain, South Africa, Italy
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Hospital St. Joseph, Marseille, FranceCompleted
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Assistance Publique - Hôpitaux de ParisCompletedGaucher DiseaseFrance
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Amicus TherapeuticsCompleted
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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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Arcturus Therapeutics, Inc.CompletedOrnithine Transcarbamylase DeficiencyNew Zealand
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Translate Bio, Inc.WithdrawnOrnithine Transcarbamylase Deficiency
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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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Horizon Therapeutics, LLCTerminatedUrea Cycle DisorderUnited States
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Horizon Pharma Ireland, Ltd., Dublin IrelandCompletedUrea Cycle DisordersUnited States, Canada
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Mark BatshawEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsTerminatedUrea Cycle DisordersUnited States, Canada
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Horizon Therapeutics, LLCCompletedUrea Cycle DisorderUnited States, Spain, Italy, Switzerland
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Yassin Abdelghaffar Charity Center for Liver Disease...Society of Friends of Liver Patients in the Arab World (SLPAW)RecruitingHereditary Tyrosinemia, Type IEgypt
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University of Missouri-ColumbiaCompleted
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University Hospital, ToursCompletedPhenylketonuriaFrance