Clinical Trials on Myopia; Refractive Error in United Kingdom
Total 2292 results
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BioMarin PharmaceuticalApproved for marketingMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVAUnited States, Puerto Rico
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Masonic Cancer Center, University of MinnesotaCompletedMucopolysaccharidosis I | Mucopolysaccharidosis VI | Mannosidosis | Mucolipidosis Type II (I-cell Disease)United States
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University of Alabama at BirminghamCompleted
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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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University of California, San DiegoBioMarin PharmaceuticalWithdrawnBehavioral Manifestations of Lesch-Nyhan DiseaseUnited States
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SanofiRecruitingFabry's DiseaseUnited States
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Horizon Pharma Ireland, Ltd., Dublin IrelandCompletedUrea Cycle DisordersUnited States, Canada
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Lysosomal and Rare Disorders Research and Treatment...Recruiting
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Millendo Therapeutics, Inc.CompletedCongenital Adrenal HyperplasiaUnited States
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Horizon Therapeutics, LLCCompletedUrea Cycle DisorderUnited States, Canada
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Genzyme, a Sanofi CompanyTerminatedGlycogen Storage Disease Type II (GSD-II) | Pompe Disease | Glycogenesis 2 Acid Maltase DeficiencyUnited States, Israel
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Ultragenyx Pharmaceutical IncCompletedWilson DiseaseUnited States, Germany
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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Canada
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4D Molecular TherapeuticsActive, not recruitingFabry DiseaseUnited States
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University of MinnesotaGenzyme, a Sanofi CompanyWithdrawnNeuropathic Pain | Fabry DiseaseUnited States
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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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ShireCompletedHunter Syndrome | Mucopolysaccharidosis II (MPS II)United States, France, United Kingdom, Germany, Sweden, Spain, Brazil, Canada, Italy, Romania
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National Institute of Neurological Disorders and...CompletedLysosomal Storage DiseaseUnited States, France, Israel, Netherlands
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Arthrosi TherapeuticsRecruitingHyperuricemia | Gout | Arthritis, Gouty | Gout ChronicUnited States, Australia, New Zealand
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Ultragenyx Pharmaceutical IncKyowa Kirin Co., Ltd.Active, not recruitingX-linked Hypophosphatemia | Hypophosphatemic RicketsUnited States, Brazil, Argentina, Canada, Chile, Colombia
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National Institutes of Health Clinical Center (CC)RecruitingCongenital Adrenal HyperplasiaUnited States
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Eunice Kennedy Shriver National Institute of Child...CompletedCongenital Adrenal Hyperplasia (CAH)United States
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Amicus TherapeuticsEngage Health Inc.CompletedFabry DiseaseUnited States
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Cyprium Therapeutics, Inc.Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompletedMenkes Disease | Occipital Horn Syndrome | Unexplained Copper DeficiencyUnited States
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Baylor Research InstituteNational Institute of Neurological Disorders and Stroke (NINDS)Completed
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ActelionCompleted
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Pacific Parkinson's Research CentreUniversity of British Columbia; Oregon Health and Science University; University... and other collaboratorsRecruitingParkinson Disease | Gaucher Disease | GBA Gene MutationUnited States, Canada
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Emalex Biosciences Inc.Psyadon PharmaTerminatedLesch-Nyhan Disease | Self-injurious BehaviorSpain, United States
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OrphalanActive, not recruiting
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Talaris Therapeutics Inc.Duke UniversityNo longer availableMetachromatic LeukodystrophyUnited States
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Genzyme, a Sanofi CompanyCompletedPompe DiseaseBelgium, United States, Czechia, Germany, Italy, Taiwan
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Denali Therapeutics Inc.Enrolling by invitationMucopolysaccharidosis IIUnited States, Canada, Belgium, Czechia, Netherlands
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Mayo ClinicCompleted
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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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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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Adrenas Therapeutics IncRecruitingCongenital Adrenal HyperplasiaUnited States
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Genzyme, a Sanofi CompanyCompletedCerebroside Lipidosis Syndrome | Glucosylceramide Beta-Glucosidase Deficiency Disease | Gaucher Disease Type I | Clucocerebrosidase Deficiency Disease | Gaucher Disease, Non-Neuronopathic FormUnited States
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Neurocrine BiosciencesCompletedCAH - Congenital Adrenal HyperplasiaUnited 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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Genzyme, a Sanofi CompanyCompletedGaucher DiseaseUnited States, Sweden, India, Netherlands, Austria, Japan, China, Serbia, France, Croatia, Australia, Portugal, Canada, Brazil, Greece, Romania, Russian Federation
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TakedaTakeda Development Center Americas, Inc.Active, not recruitingHunter SyndromeUnited States, Germany, Malaysia, Philippines, Thailand, Dominican Republic, Serbia, Vietnam
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Masonic Cancer Center, University of MinnesotaCompletedNiemann-Pick Disease | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | GM1 Gangliosidosis | Fucosidosis | Globoid Cell Leukodystrophy | Gaucher's Disease | Tay Sachs Disease | Sandhoff DiseaseUnited States
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University of California, San FranciscoRecruitingMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI | Wolman Disease | Pompe Disease Infantile-Onset | Mucopolysaccharidosis IV A | Mucopolysaccharidosis VII | Neuronopathic Gaucher DiseaseUnited States
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Cyclo Therapeutics, Inc.Active, not recruitingNiemann-Pick Disease, Type C1United States, Taiwan, United Kingdom, Israel, Spain, Brazil, Italy, Australia, Turkey, Saudi Arabia, Argentina, Poland, Germany
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Homology Medicines, IncWithdrawnMucopolysaccharidosis IIUnited States, Canada
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National MPS SocietyMediResource Inc.TerminatedMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidoses | Mucopolysaccharidosis VI | Mucopolysaccharidosis IVUnited States