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Clinical Trials on Urea Cycle Disorders, Inborn in New Zealand
Total 1095 results
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia AIndia
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PfizerCompletedHemophilia AUnited States, Argentina, Austria, Colombia, Croatia, Jordan, Mexico, New Zealand, Oman, Peru, Poland, Romania, Turkey
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Weill Medical College of Cornell UniversityWithdrawnVon Willebrand DiseaseUnited States
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Rockefeller UniversityNational Heart, Lung, and Blood Institute (NHLBI)RecruitingGlanzmann ThrombastheniaUnited States
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University of RochesterRecruitingBatten Disease | Neuronal Ceroid Lipofuscinosis | Neuronal Ceroid Lipofuscinosis CLN6 | Neuronal Ceroid Lipofuscinosis CLN3 | Neuronal Ceroid Lipofuscinosis CLN5 | Neuronal Ceroid Lipofuscinosis CLN1 | Neuronal Ceroid Lipofuscinosis CLN2 | Neuronal Ceroid Lipofuscinosis CLN7 | Neuronal Ceroid Lipofuscinosis... and other conditionsUnited States
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Wyeth is now a wholly owned subsidiary of PfizerCompleted
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National Eye Institute (NEI)CompletedNiemann Pick DiseasesUnited States, United Kingdom
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Kyowa Hakko Kirin Pharma, Inc.Kyowa Kirin Co., Ltd.CompletedX-linked HypophosphatemiaUnited States, Canada
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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Brazil
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Homology Medicines, IncWithdrawnMucopolysaccharidosis IIUnited States, Canada
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Yale UniversityWilson Disease AssociationRecruiting
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Alexion PharmaceuticalsTerminatedLysosomal Acid Lipase DeficiencyUnited States
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Yale UniversityTourette Association of AmericaCompletedTourette SyndromeUnited States
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Amicus TherapeuticsApproved for marketingPhysician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry DiseaseFabry DiseaseUnited States, Australia
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Amicus TherapeuticsCompletedFabry DiseaseUnited States
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Cerenis Therapeutics, SACompletedHomozygous Familial HypercholesterolemiaUnited States, Canada, Italy, Netherlands, United Kingdom
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Kyowa Hakko Kirin Pharma, Inc.Kyowa Kirin Co., Ltd.CompletedX-linked HypophosphatemiaUnited States, Canada
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Amicus TherapeuticsCompleted
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BayerCompleted
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University of RochesterNot yet recruitingAge-Related Macular Degeneration | Retinitis Pigmentosa | Retinal Dystrophies | Alcohol Use Disorder | Stargardt DiseaseUnited States
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsUnited States
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Maria I. NewNational Center for Research Resources (NCRR); Office of Rare Diseases (ORD)Completed21-hydroxylase DeficiencyUnited States, Brazil
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Ultragenyx Pharmaceutical IncCompletedWilson DiseaseUnited States, Germany
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Aspa TherapeuticsRecruiting
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KemPharm Denmark A/STerminatedGaucher Disease, Type 1 | Gaucher Disease, Type 3India
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Hoffmann-La RocheCompletedDown SyndromeUnited States, Spain, Canada, New Zealand, Argentina, Mexico, Singapore
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Healthcare Innovation Technology LabBayerUnknown
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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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La Jolla Pharmaceutical CompanyPRA Health SciencesCompletedHereditary HemochromatosisUnited States, Australia, France, United Kingdom
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AVROBIOTerminatedGaucher DiseaseUnited States, Canada
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors | Haemophilia A | Haemophilia BUnited States
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TakedaRecruiting
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Grifols Therapeutics LLCInstituto Grifols, S.A.CompletedCongenital AfibrinogenemiaIndia, United States, Italy, Lebanon
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Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-Linked HypophosphatemiaUnited States
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Passage Bio, Inc.Active, not recruitingGM1 Gangliosidosis | GM1 Gangliosidosis, Type I | GM1 Gangliosidosis, Type 2 | Beta-Galactosidase-1 (GLB1) DeficiencyUnited States, Canada, United Kingdom, Turkey, Brazil
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ShireApproved for marketingGaucher Disease, Type 1United States
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Cortria CorporationMontreal Heart InstituteCompletedHypertriglyceridemia | Mixed HyperlipidemiaCanada
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Aegerion Pharmaceuticals, Inc.Active, not recruitingGeneralized LipodystrophyUnited States
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PTC TherapeuticsEnrolling by invitationFriedreich AtaxiaUnited States, Australia, Brazil, Canada, France, Germany, Italy, New Zealand, Spain
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PTC TherapeuticsCompletedFriedreich AtaxiaUnited States, Australia, Brazil, Canada, France, Germany, Italy, New Zealand, Spain
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Aspa TherapeuticsRecruitingCanavan DiseaseUnited States
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AlexionCompletedWilson DiseaseUnited States, Germany, Austria, Poland, United Kingdom
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsUnited States
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Regeneron PharmaceuticalsCompletedHomozygous Familial HypercholesterolemiaUnited States, Netherlands, Australia, Austria, Canada, France, Greece, Italy, Japan, South Africa, Ukraine
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NYU Langone HealthNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)CompletedCystinuria | Primary Hyperoxaluria | Dent Disease | Adenine Phosphoribosyl Transferase DeficiencyUnited States, Iceland
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Kastle Therapeutics, LLCIonis Pharmaceuticals, Inc.CompletedMetabolic Diseases | Congenital Abnormalities | Infant, Newborn, Diseases | Genetic Diseases, Inborn | Dyslipidemias | Metabolism, Inborn Errors | Lipid Metabolism Disorders | Hypercholesterolemia | Hyperlipidemias | Hyperlipoproteinemias | Lipid Metabolism, Inborn Errors | Hyperlipoproteinemia Type II | Metabolic... and other conditionsUnited States, Canada, Brazil, Taiwan, Singapore, South Africa, United Kingdom
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Montefiore Medical CenterGenentech, Inc.Not yet recruitingHemophilia AUnited States
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New York Medical CollegeCompletedLymphoma | Leukemia | Sickle Cell Disease | Histiocytosis | Immunodeficiencies | Inborn Errors of Metabolism | Bone Marrow Failure | Beta ThalassemiaUnited States
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Michio HiranoSanthera PharmaceuticalsCompletedMELAS SyndromeUnited States
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CSL BehringRecruiting