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Clinical Trials on Purine-Pyrimidine Metabolism, Inborn Errors in New Zealand
Total 697 results
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SanofiCompletedSphingomyelin LipidosisItaly, Czechia, Belgium, France, Portugal, Turkey, United States, Argentina, Brazil, Chile, Germany, Romania, Spain
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BioMarin PharmaceuticalCompletedMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IV AUnited States, Canada, France, Taiwan, Argentina, Colombia, Spain, Turkey, Japan, Saudi Arabia, Netherlands, Denmark, Korea, Republic of, Brazil, United Kingdom, Germany, Norway, Portugal, Italy, Qatar
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University of Texas Southwestern Medical CenterNational Heart, Lung, and Blood Institute (NHLBI); Columbia University; The Cleveland... and other collaboratorsRecruitingAmyloidosis, Familial | Amyloidosis, Hereditary | Amyloidosis Cardiac | Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin Gene MutationUnited States
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Audentes TherapeuticsCompletedCrigler-Najjar SyndromeIsrael, United States, United Kingdom
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TakedaAvailableHunter SyndromeUnited States, Australia, Mexico, Spain, United Kingdom
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National Niemann-Pick Disease FoundationIcahn School of Medicine at Mount Sinai; Engage Health Inc.CompletedNiemann-Pick DiseasesUnited States
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Genzyme, a Sanofi CompanyTerminatedNiemann-Pick Disease | Acid Sphingomyelinase DeficiencyUnited States
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SanofiRecruitingFabry DiseaseChina, Finland, Germany, Norway, United Kingdom, United States, Denmark, Austria, Greece, Romania, Argentina, Brazil, Canada, Japan, Mexico, Poland, Italy, France, Turkey, Australia, Switzerland
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Audentes TherapeuticsTerminatedCrigler-Najjar SyndromeIsrael, United States, United Kingdom
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Cyclo Therapeutics, Inc.CompletedNiemann-Pick Disease, Type C1United States
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TakedaTakeda Development Center Americas, Inc.RecruitingHunter Syndrome | Mucopolysaccharidosis (MPS)United States
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Aldeyra Therapeutics, Inc.CompletedSjogren-Larsson SyndromeUnited States
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Denali Therapeutics Inc.RecruitingMucopolysaccharidosis IIUnited States, Spain, United Kingdom, Czechia, France, Canada, Italy, Belgium, Netherlands, Germany, Argentina, Sweden, Turkey, Colombia, Brazil
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NYU Langone HealthCompletedCystinuriaUnited States
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NYU Langone HealthNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Completed
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Akcea TherapeuticsRecruitingHereditary Transthyretin Amyloidosis With PolyneuropthyUnited States, Canada
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Michio HiranoSanthera PharmaceuticalsCompletedMELAS SyndromeUnited States
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedMucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type VI | Mucopolysaccharidosis Type IV | Mucopolysaccharidosis Type VIIUnited States, Canada
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Spruce BiosciencesRecruitingCongenital Adrenal Hyperplasia | 21-OHDUnited States
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Luminex CorporationCompletedAdrenal Hyperplasia, Congenital | Cystic Fibrosis | Congenital HypothyroidismUnited States
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Sangamo TherapeuticsActive, not recruitingHemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis IIUnited States
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Mayo ClinicRecruitingCystinuria | Hyperoxaluria | Dent Disease | Lowe Syndrome | Adenine Phosphoribosyltransferase DeficiencyUnited States, Canada, Iceland, Israel
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyEnrolling by invitationKidney Diseases | Urologic Diseases | Genetic Disease | Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 2 (PH2) | Primary Hyperoxaluria Type 3 (PH3)United States, France, Germany, Japan, Lebanon, Spain, United Kingdom, Australia, Canada, Italy, Netherlands, Norway, Turkey
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyRecruitingEnd Stage Renal Disease | Primary Hyperoxaluria Type 2 | Primary Hyperoxaluria Type 1Germany, Spain, United States, United Kingdom, Italy, United Arab Emirates, Lebanon, Morocco, France, Romania
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Neurocrine BiosciencesActive, not recruitingCongenital Adrenal HyperplasiaUnited States, United Kingdom, France, Greece, Italy, Spain, Belgium, Canada, Germany, Poland
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Spruce BiosciencesActive, not recruitingCongenital Adrenal HyperplasiaUnited States, Australia, Brazil, Estonia, Germany, Korea, Republic of, Latvia, Lithuania, Poland, Romania, Spain, Sweden, Turkey, Canada, Italy, United Kingdom
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Vanderbilt UniversityNational Heart, Lung, and Blood Institute (NHLBI); University of South Florida and other collaboratorsCompletedHermansky Pudlak SyndromeUnited States
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ZevraDenmarkAvailableNiemann-Pick Disease, Type CUnited States
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Bellus Health IncNational Institute of Neurological Disorders and Stroke (NINDS)CompletedStroke | Neurologic Diseases, GeneralUnited States
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Revive Therapeutics, Ltd.Unknown
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PfizerCompletedHereditary Transthyretin Amyloidosis (ATTRv) Cardiomyopathy (CM), Mixed PhenotypeUnited States
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PfizerCompletedCerebral Amyloid AngiopathyCanada, United States, Netherlands, France, United Kingdom
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Boston UniversityNational Institute of Neurological Disorders and Stroke (NINDS); Food and Drug...CompletedFamilial Amyloid Polyneuropathy | Familial AmyloidosisUnited States, Italy, Japan, Sweden, United Kingdom
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Alnylam PharmaceuticalsActive, not recruitingTransthyretin Amyloidosis (ATTR) With CardiomyopathyUnited States, Australia, Brazil, Bulgaria, France, Italy, Japan, Mexico, Netherlands, Taiwan, United Kingdom, Belgium, Czechia, Denmark, New Zealand, Sweden, Korea, Republic of, Argentina, Chile, Hong Kong, Portugal
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Intellia TherapeuticsNot yet recruitingHereditary AngioedemaFrance, New Zealand, United Kingdom
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ShireCompletedHunter Syndrome | Mucopolysaccharidosis II (MPS II)United States, France, United Kingdom, Germany, Sweden, Spain, Brazil, Canada, Italy, Romania
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Neurocrine BiosciencesActive, not recruitingCongenital Adrenal HyperplasiaUnited States, Spain, Belgium, Austria, Bulgaria, Canada, Czechia, France, Germany, Greece, Israel, Italy, Netherlands, Poland, Portugal, Serbia, Sweden, United Kingdom
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Ionis Pharmaceuticals, Inc.CompletedAmyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | TransthyretinUnited States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
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Tisento TherapeuticsChildren's Hospital of PhiladelphiaTerminated
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PfizerCompletedTransthyretin Amyloidosis | Transthyretin Gene MutationsUnited States, France, Spain, Taiwan, Israel, Korea, Republic of, Denmark, Canada, Netherlands, Japan, Sweden, Brazil, Mexico, Argentina, Portugal, Belgium, Bulgaria, Cyprus, Germany, Italy, Malaysia, Romania, Saudi Arabia, Turkey, United...
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Alnylam PharmaceuticalsActive, not recruitingTransthyretin Amyloidosis | Amyloidosis, HereditaryUnited States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Cyprus, France, Germany, Greece, Italy, Japan, Korea, Republic of, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Taiwan, United Kingdom
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Akcea TherapeuticsUnited BioSource, LLCRecruitingHereditary Transthyretin Amyloidosis With PolyneuropthyUnited States, France, Greece, Italy, Spain, Canada, Germany, Portugal
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Tisento TherapeuticsNot yet recruitingMitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)United States, Australia, Italy, Germany, Canada, United Kingdom
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Albert Einstein College of MedicineEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationMucopolysaccharidosis II | Fabry Disease | Niemann-Pick Disease, Type C | Mucopolysaccharidosis VI | Lysosomal Acid Lipase Deficiency | Metachromatic Leukodystrophy | Gaucher Disease | Cerebrotendinous Xanthomatosis | Mucopolysaccharidosis IV A | GM1 Gangliosidosis | Mucopolysaccharidosis VII | Acid Sphingomyelinase... and other conditionsUnited States
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Novartis PharmaceuticalsCompletedMyelodysplastic Syndromes | Fanconi Syndrome | Anemia, Aplastic | Beta-thalassemia | Anemia, Diamond-BlackfanUnited States
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New York Medical CollegeCompletedMucopolysaccharidosis I | Myelodysplastic Syndrome | Acute Lymphocytic Leukemia | Acute Myelogenous Leukemia | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Fucosidosis | Gaucher... and other conditionsUnited States
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Memorial Sloan Kettering Cancer CenterNational Cancer Institute (NCI); Miltenyi Biotec, Inc.Completed
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Alnylam PharmaceuticalsCompletedAmyloid Neuropathies | Amyloid Neuropathies, Familial | Amyloidosis, Hereditary | Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) | Amyloidosis, Hereditary, Transthyretin-Related | Familial Transthyretin Cardiac AmyloidosisUnited States, France, Italy, Spain, Canada, Germany, Belgium, Sweden, United Kingdom