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Clinical Trials on Purine-Pyrimidine Metabolism, Inborn Errors in New Zealand
Total 697 results
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AlexionCompletedWolman Disease | Lysosomal Acid Lipase DeficiencyUnited States, Canada, United Kingdom, Italy, France
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IntraBio IncCompletedGM2 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Germany, Spain, United Kingdom
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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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ShireCompletedGaucher DiseaseUnited States, Spain, United Kingdom, Israel
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Amicus TherapeuticsCompletedGaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher DiseaseUnited States
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Italy, Canada, France, United Kingdom, Austria, Germany
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New York Stem Cell Foundation Research InstituteSilverstein FoundationRecruitingHealthy | Parkinson Disease | Gaucher Disease | GBA Gene MutationUnited States
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OrphalanRecruiting
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Ultragenyx Pharmaceutical IncRecruitingOTC DeficiencyUnited States, Portugal, Australia, Italy, Spain, United Kingdom, Japan, Canada, Germany, France, Argentina, Netherlands, Brazil
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Kyowa Kirin, Inc.CompletedX-Linked HypophosphatemiaUnited States
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ShireCompletedGaucher DiseaseUnited States, Israel, United Kingdom, Poland, Spain
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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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LYSOGENEActive, not recruitingMucopolysaccharidosis Type IIIAGermany, United States, United Kingdom, France, Netherlands
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Macfarlane Burnet Institute for Medical Research...PATH; UNITAID; University of Melbourne; Menzies School of Health Research; Medicines... and other collaboratorsRecruitingVivax Malaria | G6PD DeficiencyPapua New Guinea
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Novartis PharmaceuticalsRecruitingHeterozygous or Homozygous Familial HypercholesterolemiaNetherlands, Israel, Hungary, Italy, Germany, Spain, United States, France, Norway, Russian Federation, South Africa, Turkey, United Kingdom, Canada, Switzerland, Brazil, Lebanon, Slovenia, Poland, Taiwan, Malaysia
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ActelionCompletedGaucher Disease Type 1United States, Taiwan, Canada, Spain, Australia, France, Brazil, Hungary, Czechia, Germany, Italy, Netherlands, United Kingdom
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Ultragenyx Pharmaceutical IncActive, not recruitingOrnithine Transcarbamylase (OTC) DeficiencyCanada, United States, France, Spain, United Kingdom
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SanofiRecruitingGlycogen Storage Disease Type IIBelgium, United States, China, Germany, Italy, Netherlands, Spain, Taiwan, United Kingdom
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Kaleido BiosciencesTerminatedUrea Cycle DisorderBelgium, United States, Spain, United Kingdom, Germany, Switzerland, Turkey
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Genzyme, a Sanofi CompanyActive, not recruitingGaucher Disease Type 1 | Gaucher Disease Type 3Germany, United States, Japan, United Kingdom
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Horizon Therapeutics, LLCCompletedUrea Cycle DisorderUnited States, Canada
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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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Novartis PharmaceuticalsCompletedElevated Cholesterol | Homozygous Familial Hypercholesterolemia | Heterozygous Familial Hypercholesterolemia | ASCVDUnited States, Canada, Czechia, Denmark, Germany, Hungary, Netherlands, Poland, South Africa, Spain, Sweden, Ukraine, United Kingdom
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase Deficiency | LAL-Deficiency | Cholesterol Ester Storage Disease (CESD)France, United Kingdom, United States, Canada, Czechia
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SanofiRecruitingFabry's DiseaseUnited States
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SanofiRecruitingGlycogen Storage Disease Type IIBelgium, United States, Italy, Spain, Taiwan, United Kingdom, Netherlands, France, Germany
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Genzyme, a Sanofi CompanyCompletedPompe Disease | Glycogen Storage Disease Type II (GSD II)United States, Russian Federation, United Kingdom, Bulgaria, India, Ukraine
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Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-linked HypophosphatemiaUnited States, Korea, Republic of, Canada, Japan, France, Denmark
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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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Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-Linked HypophosphatemiaUnited States
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Allena PharmaceuticalsCompletedEnteric Hyperoxaluria | Primary Hyperoxaluria | HyperoxalemiaUnited States, United Kingdom, Germany
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Ionis Pharmaceuticals, Inc.Active, not recruitingFamilial Chylomicronemia SyndromeSpain, United Kingdom, France, Canada, United States, Italy, Netherlands, Norway, Portugal, Slovakia, Sweden
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Freeline TherapeuticsRecruitingGaucher Disease, Type 1Spain, Israel, United States, United Kingdom, Brazil, Germany, Paraguay
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Ultragenyx Pharmaceutical IncCompletedMucopolysaccharidosis | Sly Syndrome | Mucopolysaccharidosis VII | MPS VIISpain, United States, Portugal
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Amicus TherapeuticsTerminatedLate-onset Pompe DiseaseUnited States, Canada, Australia, Belgium
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Ultragenyx Pharmaceutical IncCompletedGlucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)United States, Spain, Australia, France, Israel, Italy, United Kingdom
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Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-linked HypophosphatemiaUnited States, Korea, Republic of, Japan, Ireland, United Kingdom, France, Italy
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BioMarin PharmaceuticalCompletedMucopolysaccharidosis IVA (Morquio A Syndrome)Australia
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Genzyme, a Sanofi CompanyTerminatedPompe DiseaseUnited States
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LIB Therapeutics LLCMedpace, Inc.Enrolling by invitationFamilial Hypercholesterolemia | Elevated Cholesterol | Cardiovascular Disease With Mention of ArteriosclerosisUnited States, Israel, Norway, South Africa, Turkey, India
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Ionis Pharmaceuticals, Inc.Akcea TherapeuticsCompletedFamilial Chylomicronemia SyndromeUnited States, Brazil, Canada, France, Germany, Hungary, Israel, Italy, Netherlands, South Africa, Spain, United Kingdom
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National Center for Research Resources (NCRR)Icahn School of Medicine at Mount SinaiCompletedMetabolism, Inborn Errors | Leukodystrophy, Globoid Cell | Tay-Sachs Disease | Porphyria, ErythropoieticUnited 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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BioMarin PharmaceuticalApproved for marketingMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVAUnited States, Puerto Rico
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Ionis Pharmaceuticals, Inc.Akcea TherapeuticsCompletedFamilial Chylomicronemia SyndromeUnited States, Israel, Spain, Italy, United Kingdom, Netherlands, Canada, Hungary, Sweden, France, Norway, Portugal, Slovakia
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ActelionCompleted
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Genzyme, a Sanofi CompanyRecruitingMucopolysaccharidosis I (MPS I)Croatia, United States, Argentina, Australia, Belgium, Brazil, Canada, Denmark, France, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Lebanon, Malaysia, Netherlands, Pakistan, Philippines, Poland, Saudi Arabia, Singapore, Slovaki... and more
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Yale UniversityWilson Disease AssociationRecruiting
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Passage Bio, Inc.SuspendedLeukodystrophy, Globoid CellUnited States, Brazil, Netherlands, United Kingdom, Canada, Israel