- ICH GCP
- US Clinical Trials Registry
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Clinical Trials on Brain Diseases, Metabolic
Total 382163 results
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Ultragenyx Pharmaceutical IncActive, not recruitingOrnithine Transcarbamylase (OTC) DeficiencyCanada, United States, France, Spain, United Kingdom
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OrphalanErgomedRecruitingWilson's DiseaseSpain, United Kingdom, Belgium, Germany, Poland, France, Saudi Arabia
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OrphalanCompletedWilson's DiseaseUnited Kingdom
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University College, LondonUnknown
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NobelpharmaCompleted
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Medical University of ViennaRecruitingPompe Disease | McArdle DiseaseAustria
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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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University of LausanneUniversity of Modena and Reggio EmiliaCompletedN-Acetylneuraminic Acid Storage DiseaseSwitzerland, Italy
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Assistance Publique - Hôpitaux de ParisRecruiting
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ShireTakeda Development Center Americas, Inc.Active, not recruitingMetachromatic Leukodystrophy (MLD)United States, Canada, Belgium, Israel, Netherlands, United Kingdom, Germany, Spain, Japan, Brazil, Argentina, France, Greece, Italy
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TakedaWithdrawnMetachromatic Leukodystrophy (MLD)Spain
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ShireCompletedMetachromatic Leukodystrophy (MLD)Denmark
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ShireCompletedLate Infantile Metachromatic LeukodystrophyDenmark
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Assistance Publique - Hôpitaux de ParisEuropean Leukodystrophy AssociationCompletedLate Infantile Metachromatic LeukodystrophyFrance
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Lysosomal and Rare Disorders Research and Treatment...UnknownParkinson Disease | Gaucher DiseaseUnited States
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Late Onset Tay-Sachs DiseaseUnited States
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University Children's Hospital, ZurichSanofiCompleted
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McGill University Health Centre/Research Institute...RecruitingMitochondrial Diseases | Mitochondrial Encephalomyopathy | Mitochondrial Encephalopathy | Mitochondrial DNA Depletion | Mitochondrial Metabolism DisordersCanada
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Vitaflo International, LtdUniversity College London HospitalsRecruiting
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AmgenUcyclyd Pharma, Inc.CompletedHepatic Encephalopathy | Urea Cycle DisordersUkraine
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AceLink Therapeutics, Inc.Tigermed Consulting Co., LtdRecruiting
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Albina Nowak, MDNot yet recruiting
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Chiesi Farmaceutici S.p.A.ICON plcRecruiting
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Children's Hospital Medical Center, CincinnatiSanofiRecruitingFabry DiseaseUnited States
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University of Sao Paulo General HospitalPfizerRecruiting
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Genzyme, a Sanofi CompanyRecruitingFabry DiseaseCroatia, United States, Argentina, Australia, Belgium, Bulgaria, Canada, Germany, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Malaysia, Philippines, Poland, Romania, Singapore, Slovakia, Taiwan, Thailand, United Kingdom, Vietn... and more
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ShireTerminatedFabry DiseaseSpain, Greece, Germany, Portugal, Canada, Finland, Poland, Sweden
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Amicus Therapeutics France SASCompleted
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Sangamo TherapeuticsActive, not recruitingFabry DiseaseUnited States, United Kingdom, Taiwan, Australia, Germany, Italy, Canada
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Vastra Gotaland RegionKarolinska University Hospital; Göteborg University; Uppsala University HospitalCompleted
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4D Molecular TherapeuticsActive, not recruitingFabry DiseaseUnited States
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Freeline TherapeuticsCompletedLysosomal Storage Diseases | Fabry DiseaseGermany, United Kingdom
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Serbia, Spain, United Kingdom, Australia, Paraguay
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AVROBIOTerminatedFabry DiseaseAustralia, Brazil
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Chiesi Farmaceutici S.p.A.Chiesi USA, Inc.AvailableFabry DiseaseUnited States
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Idorsia Pharmaceuticals Ltd.CompletedFabry DiseaseUnited States, Spain, Belgium, Australia, Austria, Canada, Germany, Ireland, Italy, Netherlands, Norway, Poland, Switzerland, United Kingdom
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ShireCompleted
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ShireCompletedFabry DiseaseUnited States, Slovenia, Australia, Finland, Poland, Czechia, United Kingdom
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University Hospital MuensterAmicus TherapeuticsCompleted
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Genzyme, a Sanofi CompanyCompletedFabry DiseaseFrance, Poland, United States, Russian Federation, United Kingdom
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Amicus TherapeuticsCompletedFabry DiseaseCanada, Belgium, United States, Australia, Netherlands
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Idorsia Pharmaceuticals Ltd.Completed
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Greenovation Biotech GmbHFGK Clinical Research GmbHCompleted
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Université de MontréalGenzyme, a Sanofi CompanyCompleted
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Chang Gung Memorial HospitalSanofiUnknown
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Genzyme, a Sanofi CompanyCompletedFabry DiseaseUnited States, Poland, Netherlands, Canada, Argentina, United Kingdom, Norway, Brazil, Czech Republic
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Genzyme, a Sanofi CompanyCompleted
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Genzyme, a Sanofi CompanyCompletedFabry DiseaseCzech Republic, Estonia, Poland, Slovakia
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Genzyme, a Sanofi CompanyCompletedFabry DiseaseUnited States, France, Poland, United Kingdom