Clinical Trials on Purine-Pyrimidine Metabolism, Inborn Errors in New Zealand
Total 697 results
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SanofiRegeneron PharmaceuticalsCompletedHypercholesterolemiaUnited States, Austria, Canada, Czech Republic, Denmark, France, Israel, Netherlands, Norway, Russian Federation, South Africa, Spain, Sweden, United Kingdom
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Amicus TherapeuticsCompleted
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PfizerTerminatedHyperlipoproteinemia Type IIICanada, United States
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BioMarin PharmaceuticalCompleted
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Ritter Pharmaceuticals, Inc.CompletedLactose IntoleranceUnited States
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Travere Therapeutics, Inc.UnknownCerebrotendinous Xanthomatosis (CTX)United States
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Canada, United Kingdom, Portugal
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Glycogenosis 2United States
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SanofiCompletedOsteochondrodysplasiaUnited States
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Dicerna Pharmaceuticals, Inc.CompletedKidney Diseases | Urologic Diseases | Genetic Disease | Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 2 (PH2)Poland, United Kingdom, New Zealand, United States, Australia, Canada, France, Germany, Israel, Italy, Japan, Lebanon, Netherlands, Romania, Spain
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PfizerCompletedHypertriglyceridemia | Hyperlipoproteinemia Type IVUnited States, Canada
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BioMarin PharmaceuticalCompletedPhenylketonuria (PKU)United States
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State University of New York at BuffaloEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsRecruiting
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The Cooper Health SystemCompletedMetachromatic LeukodystrophyUnited States
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Regeneron PharmaceuticalsCompletedHomozygous Familial HypercholesterolemiaUnited States, Netherlands, South Africa, Australia, Austria, Canada, Czechia, France, Greece, Italy, Japan, Ukraine
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Homology Medicines, IncTerminated
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LIB Therapeutics LLCCompletedHomozygous Familial HypercholesterolemiaUnited States, Israel, Norway, South Africa, Turkey, India
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Intellia TherapeuticsRecruitingTransthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac AmyloidosisUnited Kingdom, New Zealand, Sweden
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Amryt PharmaRecruitingHomozygous Familial HypercholesterolemiaItaly, United States, Argentina, Canada, France, Greece, Netherlands, United Kingdom
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Arcturus Therapeutics, Inc.Active, not recruitingOrnithine Transcarbamylase DeficiencyUnited States
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AVROBIOTerminatedType 1 Gaucher DiseaseUnited States, Canada
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AmgenCompletedFamilial HypercholesterolemiaCanada, Italy, Norway, Belgium, United States, Turkey, Australia, Austria, South Africa, Poland, Switzerland, Spain, Portugal, Slovenia, Brazil, Russian Federation, Hungary, United Kingdom, Colombia, Czechia, Netherlands, Greece, Ma...
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Jnana TherapeuticsRecruitingPhenylketonuriaUnited States, Australia
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Pompe Disease Infantile-OnsetUnited States, France, South Africa
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BioMarin PharmaceuticalRecruitingPhenylketonuria (PKU)United States, Germany, Italy
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Weill Medical College of Cornell UniversityNational Institute of Neurological Disorders and Stroke (NINDS); National Institutes... and other collaboratorsCompletedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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Weill Medical College of Cornell UniversityTerminatedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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PfizerCompletedHyperlipidemia | Hypercholesterolemia, FamilialUnited States, Canada, South Africa, Czech Republic, Italy, Finland, Netherlands, France
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Amicus TherapeuticsTerminatedBatten Disease | Neuronal Ceroid Lipofuscinosis CLN6 | Neuronal Ceroid Lipofuscinosis CLN3United States
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Horizon Pharma Ireland, Ltd., Dublin IrelandCompletedUrea Cycle DisordersUnited States
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Neurogene Inc.RecruitingNeuronal Ceroid Lipofuscinosis CLN5United States, United Kingdom
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGaucher Disease Type 1United States
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Andrea GropmanEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsRecruitingBrain Diseases, Metabolic, Inborn | Amino Acid Metabolism, Inborn Errors | Urea Cycle DisordersUnited States, Canada, Germany, Switzerland
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PfizerTerminatedMixed HyperlipidemiaUnited States, Canada, Czech Republic, Finland, France, Netherlands
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Recordati Rare DiseasesTarget PharmaSolutions, Inc.RecruitingPropionic Acidemia | Methylmalonic Acidemia | HyperammonemiaUnited States
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Ultragenyx Pharmaceutical IncTerminatedGlucose Transporter Type 1 Deficiency SyndromeUnited States, Spain, Denmark, United Kingdom, Australia
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HemoShear TherapeuticsTerminatedPropionic Acidemia | Methylmalonic AcidemiaUnited States, Saudi Arabia, Australia
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Nacuity Pharmaceuticals, Inc.Recruiting
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Horizon Pharma Ireland, Ltd., Dublin IrelandCompletedUrea Cycle DisordersUnited States, Canada
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Regeneron PharmaceuticalsSanofiCompletedHomozygous Familial HypercholesterolemiaUnited States, South Africa, Canada, Greece, Japan, Ukraine, Austria, Czechia, France, Germany, Italy, Taiwan, Turkey
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Horizon Therapeutics, LLCCompletedUrea Cycle DisorderUnited States, Spain, Italy, Switzerland
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PTC TherapeuticsRecruitingPhenylketonuriaUnited States, Germany, Spain, Australia, Brazil, United Kingdom, Denmark, Mexico, Portugal, Turkey, Canada, Georgia, Italy, Netherlands, Japan
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Travere Therapeutics, Inc.Active, not recruiting
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Novartis PharmaceuticalsActive, not recruitingFamilial Hypercholesterolemia - HeterozygousUnited States, Germany, South Africa, Greece, Italy, Czechia, Taiwan, Slovakia, Turkey, France, Russian Federation, Canada, Spain, Jordan, Switzerland, Hungary, Poland, Netherlands, Norway, United Kingdom, Lebanon, Argentina, Brazil, ... and more
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PfizerCompletedGaucher DiseaseUnited States, Chile, United Kingdom, Israel, Canada, Spain, South Africa, Italy
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Kyowa Hakko Kirin Pharma, Inc.Completed
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Merck Sharp & Dohme LLCActive, not recruitingHypercholesterolemia | Familial HypercholesterolemiaColombia, Israel, Taiwan, United States, Argentina, China, Germany, Italy, Japan, Korea, Republic of, Mexico, South Africa, Spain, Turkey
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PfizerCompletedGaucher DiseaseUnited Kingdom, Canada, Israel, United States, Australia, Spain
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BioMarin PharmaceuticalCompletedPhenylketonuria (PKU)United States
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PfizerCompletedHyperlipidemia | Hypercholesterolemia, FamilialAustralia, Canada, United States, France, South Africa, Sweden, Norway, Denmark, Iceland