Clinical Trials on Brain Diseases, Metabolic in United States
Total 147731 results
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BioMarin PharmaceuticalCompleted
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Canada, United Kingdom, Portugal
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Myrtelle Inc.RecruitingCanavan DiseaseUnited States
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University of FloridaUniversity of Miami; University of Massachusetts, WorcesterNo longer available
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Cytonet GmbH & Co. KGTerminatedUrea Cycle DisordersUnited States, Canada
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AVROBIOTerminatedGaucher DiseaseUnited States, Canada
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National Organization for Rare DisordersFood and Drug Administration (FDA)Active, not recruitingMetachromatic LeukodystrophyUnited States
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University of PittsburghActive, not recruitingMetachromatic LeukodystrophyUnited States
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ShireCompletedGaucher Disease, Type 1Spain, Israel, United States, India, Paraguay, United Kingdom, Russian Federation, Tunisia, Argentina
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Baylor Research InstituteNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsUnknown
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Amicus TherapeuticsRecruiting
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National Institute of Neurological Disorders and...CompletedFabry DiseaseUnited States
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O & O Alpan LLCCompletedFabry DiseaseUnited States
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Ohio State UniversityGenzyme, a Sanofi CompanyCompleted
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National Institute of Neurological Disorders and...Completed
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National Institute of Neurological Disorders and...Completed
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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Yale UniversityBausch Health Americas, Inc.CompletedWilson DiseaseUnited States
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Duke UniversityGenzyme, a Sanofi CompanyCompletedNeuropathy | Myopathy | Glycogen Storage Disease Type II (Late-onset Pompe Disease)United States
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AlexionCompletedWilson DiseaseUnited States
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Children's Hospital of PhiladelphiaUniversity of Wisconsin, Madison; Ionis Pharmaceuticals, Inc.; Pennsylvania Department...Recruiting
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IntraBio IncCompletedGM2 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Germany, Spain, United Kingdom
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BioMarin PharmaceuticalCompletedPhenylketonuriasUnited States
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe DiseaseUnited States
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Genzyme, a Sanofi CompanyCompletedPompe Disease Late-Onset | Glycogen Storage Disease Type II GSD IIUnited States
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StemCells, Inc.CompletedPelizaeus-Merzbacher Disease | PMDUnited States
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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Zymenex A/SCompletedInfantile Globoid Cell LeukodystrophyUnited States
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ShireCompletedGaucher DiseaseUnited States, Spain, United Kingdom, Israel
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Canada, United Kingdom, Portugal
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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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BioMarin PharmaceuticalCompletedPhenylketonuria (PKU)United States, Turkey, Canada, France, Germany, Italy, United Kingdom
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New York Stem Cell Foundation Research InstituteSilverstein FoundationRecruitingHealthy | Parkinson Disease | Gaucher Disease | GBA Gene MutationUnited States
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National Institute of Neurological Disorders and...National Institute of Neurological Disorders and Stroke@@@Shire Human Genetic...Completed
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The University of Texas Health Science Center,...RecruitingLeigh Disease | Leigh Syndrome | Leigh's Necrotizing Encephalopathy | Subacute Necrotizing Encephalomyopathy | Subacute Necrotizing EncephalomyelopathyUnited States
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type IIUnited States, Taiwan, Israel, United Kingdom, France
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Astellas Gene TherapiesRecruitingPompe Disease (Late-onset)United States, United Kingdom
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Amicus TherapeuticsCompletedGlycogen Storage Disease Type II | Pompe DiseaseUnited States
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SynlogicCompletedPhenylketonuriaUnited States
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Amicus TherapeuticsAvailablePompe Disease Infantile-OnsetTaiwan, United States, Italy
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Nutricia UK LtdCompletedPKU | TyrosinemiasUnited Kingdom
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Cedars-Sinai Medical CenterNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsUnknown
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National Institute of Neurological Disorders and...CompletedHealthy | Fabry Disease | Cerebrovascular AccidentUnited States
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University of UtahGenzyme, a Sanofi CompanyCompletedFabry DiseaseUnited States
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Exsar CorporationThe Hospital for Sick Children; NYU Langone Health; University Hospitals Cleveland...WithdrawnG(M2) Ganglioside | Tay-Sachs Disease Ganglioside | Sandhoff Disease GangliosideUnited States, Canada
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Amicus TherapeuticsCompletedGaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher DiseaseUnited Kingdom, Israel, South Africa, United States
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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Aeglea BiotherapeuticsTerminatedHomocystinuria Due to Cystathionine Beta-Synthase DeficiencyUnited States, United Kingdom, Australia
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APR Applied Pharma Research s.a.RecruitingPhenylketonuriasUnited Kingdom