- ICH GCP
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Clinical Trials on Myopia; Refractive Error in United Kingdom
Total 2295 results
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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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BioMarin PharmaceuticalActive, not recruitingLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2United States
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Horizon Pharma USA, Inc.Completed
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Eunice Kennedy Shriver National Institute of Child...RecruitingLysosomal Storage Disease | Cholesterol MetabolismUnited States
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LG ChemRecruitingHyperuricemia | Gout | Gout Flare | TophiUnited States
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University of UtahGenzyme, a Sanofi CompanyCompletedFabry DiseaseUnited States
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Georgetown UniversityCompletedEnd Stage Renal Disease | HyperhomocysteinemiaUnited States
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University of PennsylvaniaAstraZeneca; Ionis Pharmaceuticals, Inc.Active, not recruitingHereditary Amyloidosis, Transthyretin-RelatedUnited States
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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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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Pompe DiseaseUnited States
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Nuon Therapeutics, Inc.CompletedHyperuricemia | Moderate to Severe GoutUnited States
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Cedars-Sinai Medical CenterNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsUnknown
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New York Stem Cell Foundation Research InstituteSilverstein FoundationRecruitingHealthy | Parkinson Disease | Gaucher Disease | GBA Gene MutationUnited States
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Lundquist Institute for Biomedical Innovation at...TerminatedMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VIUnited States
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National Institute of Neurological Disorders and...CompletedHealthy | Fabry Disease | Cerebrovascular AccidentUnited States
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National Human Genome Research Institute (NHGRI)CompletedGeneralized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets Type2United States
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type IIUnited States, France, Germany, Israel, Italy, Netherlands, Taiwan
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Novartis PharmaceuticalsCompletedAcute Gouty Arthritis FlaresUnited States, Estonia, Lithuania, Germany, Russian Federation, Canada, Australia, Latvia, Ukraine
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Cyprium Therapeutics, Inc.Enrolling by invitation
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Eunice Kennedy Shriver National Institute of Child...Completed
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National Institute of Neurological Disorders and...CompletedFabry DiseaseUnited States
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bluebird bioTerminatedCerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)United States, United Kingdom, Argentina, Canada, Germany, Italy, Netherlands
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Horizon Therapeutics, LLCCompletedUrea Cycle DisordersUnited States, Canada
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Intellia TherapeuticsRegeneron PharmaceuticalsRecruitingTransthyretin Amyloidosis (ATTR) With CardiomyopathyUnited States, Australia, United Kingdom, New Zealand, Canada
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ArmaGen, IncCompletedMucopolysaccharidosis IIUnited States, Germany, Philippines
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bluebird bioActive, not recruitingCerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)United States, Argentina, Australia, Brazil, France, Germany, Netherlands, United Kingdom, Italy
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Office of Rare Diseases (ORD)UnknownAdrenal Hyperplasia, CongenitalUnited States, Brazil, France
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Pompe Disease Infantile-OnsetUnited States, France, South Africa
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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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Mandos LLCTerminatedNiemann-Pick Disease, Type CUnited States, Australia, United Kingdom, Turkey, Switzerland, Germany, New Zealand, Singapore, France
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Boston Children's HospitalDana-Farber Cancer Institute; Brigham and Women's Hospital; Schering-PloughCompletedProgeria | Hutchinson-Gilford SyndromeUnited States
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Children's Hospital of Orange CountyActive, not recruitingNeuronal Ceroid-Lipofuscinoses | Neuronal Ceroid Lipofuscinosis CLN2 | Spinocerebellar Ataxia, Autosomal Recessive 7United States
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Lundquist Institute for Biomedical Innovation at...CompletedMucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type VIUnited States
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Novartis PharmaceuticalsCompletedAcute Gouty ArthritisGermany, United States, Lithuania, Canada
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University of Texas Southwestern Medical CenterNational Institutes of Health Clinical Center (CC); University of Michigan; Children... and other collaboratorsNot yet recruitingCongenital Adrenal HyperplasiaUnited States
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Eunice Kennedy Shriver National Institute of Child...RecruitingCHILD Syndrome | Smith Lemli Opitz Syndrome | Lathosterolosis | DesmosterolosisUnited States
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Psyadon PharmaCompletedLesch-Nyhan DiseaseUnited States
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PfizerCompletedTransthyretin Amyloidosis Cardiomyopathy (ATTR-CM)Italy, Romania, France, Australia, Austria, Portugal, Slovakia, Slovenia, Spain, United Kingdom
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Cystinosis Research FoundationUnknownCystinosis | Nephropathic Cystinosis | Renal Fanconi SyndromeUnited States
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Amicus TherapeuticsCompletedGaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher DiseaseUnited States
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Horizon Pharma Ireland, Ltd., Dublin IrelandCompletedUrea Cycle DisordersUnited States, Canada
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Horizon Pharma USA, Inc.Completed
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BioMarin PharmaceuticalApproved for marketingMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVAUnited States, Puerto Rico
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Sangamo TherapeuticsTerminatedMucopolysaccharidosis II | MPS IIUnited States
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Horizon Pharma Rheumatology LLCCompletedRefractory Chronic GoutUnited States
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LG ChemRecruitingHyperuricemia | Gout | Gout Flare | TophiUnited States
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Novartis PharmaceuticalsCompletedAcute Gouty ArthritisHungary, Germany, United States, Lithuania, Canada
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National Center for Research Resources (NCRR)Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompletedCystinosisUnited States
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Greenwood Genetic CenterCompletedGalactosialidosis | Sialidosis | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Beta Mannosidosis | Mucolipidosis II | Mucolipidosis III | Schindler DiseaseUnited States