Clinical Trials on Purine-Pyrimidine Metabolism, Inborn Errors in New Zealand
Total 703 results
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AVROBIOTerminatedGaucher DiseaseUnited States, Canada
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PTC TherapeuticsCompletedPhenylketonuriaSpain, United States, Australia, Germany, United Kingdom, Brazil, Denmark, Canada, France, Georgia, Italy, Mexico, Netherlands, Portugal, Turkey
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PfizerCompletedHeterozygous Familial HypercholesterolemiaCanada, Netherlands, United Kingdom, United States, Spain, Poland, Finland, Norway, South Africa, Italy, Bulgaria
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University of CologneCompletedPrimary Hyperoxaluria Type IGermany
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Dicerna Pharmaceuticals, Inc.CompletedPrimary Hyperoxaluria Type 3United States, Germany, Netherlands, United Kingdom
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Horizon Pharma Ireland, Ltd., Dublin IrelandCompletedUrea Cycle DisordersUnited States, Canada
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PfizerCompletedGaucher DiseaseChile, United Kingdom, Canada, Spain, Israel, United States, Australia, South Africa
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ShireApproved for marketingGaucher Disease, Type 1United States
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BioMarin PharmaceuticalCompletedTetrahydrobiopterin Deficiencies | Hyperphenylalaninemia, Non-PhenylketonuricGermany, United States
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ModernaTX, Inc.CompletedPropionic Acidemia | Methylmalonic AcidemiaUnited States, United Kingdom, Spain, France
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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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Mark BatshawEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsTerminatedUrea Cycle DisordersUnited States, Canada
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Weill Medical College of Cornell UniversityNathan's Battle FoundationCompletedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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Yale UniversityNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)CompletedHypophosphatemic Rickets, X Linked DominantUnited States
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States, Canada
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Phoenix NestNational Institute of Neurological Disorders and Stroke (NINDS)Enrolling by invitationSanfilippo Syndrome Type DUnited States
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Amicus TherapeuticsActive, not recruitingPompe Disease (Late-onset)United States, Germany, Taiwan, Belgium, Australia, Slovenia, Spain, Netherlands, Hungary, Japan, Austria, United Kingdom, France, Argentina, Canada, Sweden, Denmark, New Zealand, Greece, Korea, Republic of, Bosnia and Herzegovina, Ita... and more
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AldagenTerminatedMucopolysaccharidosis | Inborn Errors of Metabolism | Lysosomal Storage Disorders | Inherited Metabolic Diseases | Peroxisomal Storage DiseasesUnited States
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QOL Medical, LLCJohns Hopkins University; Massachusetts General Hospital; Baylor College of Medicine and other collaboratorsCompletedCongenital Sucrase-isomaltase Deficiency (CSID)United States
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Weill Medical College of Cornell UniversityNational Institutes of Health (NIH)CompletedBatten Disease | Late-Infantile Neuronal Ceroid LipofuscinosisUnited States
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Weill Medical College of Cornell UniversityCompletedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase DeficiencyFrance, Poland, United Kingdom, Spain, Mexico, Turkey, Japan, Australia, Russian Federation, United States, Germany, Italy, Czechia, Argentina, Croatia
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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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Yale UniversityCompleted
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Yale UniversityNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)CompletedHyperparathyroidism | Hypophosphatemia, FamilialUnited States
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Ionis Pharmaceuticals, Inc.Active, not recruitingFamilial Chylomicronemia SyndromeUnited States, Canada, Sweden
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Yale UniversityBausch Health Americas, Inc.CompletedWilson DiseaseUnited States
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Amicus TherapeuticsCompletedGlycogen Storage Disease Type II | Pompe DiseaseUnited States
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Uta Lichter-KoneckiMedical College of Wisconsin; Children's National Research InstituteCompletedUrea Cycle Disorders | Organic AcidemiasUnited States
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Intellia TherapeuticsActive, not recruitingTransthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac AmyloidosisFrance, United Kingdom, New Zealand, Sweden
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Amicus TherapeuticsCompletedPompe Disease (Late-onset)United States, Australia, Korea, Republic of, Japan, Taiwan, Belgium, Slovenia, New Zealand, United Kingdom, France, Argentina, Canada, Spain, Bosnia and Herzegovina, Germany, Sweden, Hungary, Austria, Bulgaria, Denmark, Greece, I... and more
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KemPharm Denmark A/STerminatedGaucher Disease, Type 1 | Gaucher Disease, Type 3India
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ShireCompletedGaucher Disease, Type 1United States, Israel, Argentina, India, Korea, Republic of, Paraguay, Poland, Russian Federation, Spain, Tunisia, United Kingdom
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The Medicines CompanyCompletedElevated Cholesterol | Heterozygous Familial HypercholesterolemiaUnited States, Canada, Czechia, Denmark, Netherlands, South Africa, Spain, Sweden
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Aeglea BiotherapeuticsTerminatedHomocystinuria Due to Cystathionine Beta-Synthase DeficiencyUnited States, United Kingdom, Australia
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ShireCompletedGaucher Disease, Type 1Spain, Israel, United States, India, Paraguay, United Kingdom, Russian Federation, Tunisia, Argentina
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Regeneron PharmaceuticalsSanofiTerminatedHeterozygous Familial Hypercholesterolemia | Non-familial HypercholesterolemiaUnited States, Bulgaria, Estonia, Russian Federation, South Africa, Ukraine
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Amicus TherapeuticsCompleted
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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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Horizon Therapeutics, LLCCompletedUrea Cycle DisordersUnited States, Canada
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ShireActive, not recruiting
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase Deficiency | Cholesterol Ester Storage Disease(CESD) | LAL-DeficiencyFrance, United States, United Kingdom, Czechia
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AlexionCompletedWolman Disease | Lysosomal Acid Lipase DeficiencyUnited States, Canada, United Kingdom, Italy, France
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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 CompanyCompleted
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ShireCompletedGaucher DiseaseUnited States, Spain, United Kingdom, Israel
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Agios Pharmaceuticals, Inc.Active, not recruitingPediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic AnemiaUnited States, Spain, Denmark, Turkey, Netherlands, Czechia, United Kingdom, Germany, Canada, Switzerland
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IntraBio IncCompletedGM2 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Germany, Spain, United Kingdom
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Alexion Pharmaceuticals, Inc.TerminatedWilson DiseaseUnited States, Spain, Taiwan, France, Czechia, Hungary, Turkey, United Kingdom, Austria, Germany, Israel, Serbia, Australia, Japan, Denmark, Korea, Republic of, New Zealand, Canada, Russian Federation, Poland, Singapore, Hong Kong