Clinical Trials on Centronuclear Myopathy, X-Linked in United Kingdom
Total 928 results
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Eunice Kennedy Shriver National Institute of Child...Food and Drug Administration (FDA)TerminatedAdrenoleukodystrophy | AdrenomyeloneuropathyUnited States
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Sarepta Therapeutics, Inc.Hoffmann-La RocheRecruitingDuchenne Muscular DystrophyBelgium, United States, Japan, Spain, Italy, United Kingdom
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ReveraGen BioPharma, Inc.University of Pittsburgh; Newcastle University; European Union; Cooperative International...CompletedDuchenne Muscular DystrophyUnited States, Belgium, Canada, United Kingdom, Spain, Netherlands, Sweden, Czechia, Australia, Greece, Israel
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University of RochesterNational Institute of Neurological Disorders and Stroke (NINDS); Newcastle... and other collaboratorsCompletedDuchenne Muscular DystrophyUnited States, Canada, United Kingdom, Germany, Italy
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Santhera PharmaceuticalsTerminatedDuchenne Muscular DystrophyUnited States, Belgium, United Kingdom, Austria, France, Germany, Italy, Spain, Switzerland
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PfizerTerminatedDuchenne Muscular DystrophyUnited States, Canada, United Kingdom, Japan, Italy
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Emalex Biosciences Inc.Psyadon PharmaTerminatedLesch-Nyhan Disease | Self-injurious BehaviorSpain, United States
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CSL BehringTerminatedHemophilia A With Inhibitors | Hemophilia B With InhibitorsGeorgia, Italy, Malaysia, Russian Federation, South Africa, Spain, Thailand, Ukraine, United Kingdom
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NS Pharma, Inc.Nippon Shinyaku Co., Ltd.Active, not recruitingDuchenne Muscular DystrophyKorea, Republic of, Chile, Japan, China, United Kingdom, Netherlands, Spain, Norway, Russian Federation, Australia, Greece, Italy, New Zealand, Turkey, Mexico, Czechia, Canada
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Washington University School of MedicineRecruitingSevere Combined Immunodeficiency | Chronic Granulomatous Disease | DiGeorge Syndrome | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | X-linked Lymphoproliferative Syndrome | X-linked Agammaglobulinemia | Chediak-Higashi Syndrome | IPEX | Autoimmune Lymphoproliferative Syndrome | Common Variable... and other conditionsUnited States
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Masonic Cancer Center, University of MinnesotaRecruiting
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University of PittsburghNational Institute of Neurological Disorders and Stroke (NINDS); Rare Diseases...RecruitingPyruvate Dehydrogenase Complex Deficiency DiseaseUnited States
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TakedaTakeda Development Center Americas, Inc.RecruitingHunter Syndrome | Mucopolysaccharidosis (MPS)United States
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BiogenCompleted
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National MPS SocietyMediResource Inc.TerminatedMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidoses | Mucopolysaccharidosis VI | Mucopolysaccharidosis IVUnited States
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NS Pharma, Inc.Nippon Shinyaku Co., Ltd.CompletedDuchenne Muscular DystrophyKorea, Republic of, Chile, Taiwan, China, United States, United Kingdom, Netherlands, Spain, Australia, Norway, Russian Federation, Greece, Italy, Turkey, Mexico, Canada, Hong Kong, New Zealand, Ukraine
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ItalfarmacoSyneos HealthCompletedDuchenne Muscular DystrophyUnited States, Italy, Spain, Canada, Belgium, France, Germany, Israel, Netherlands, Serbia, United Kingdom
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PfizerTerminatedDuchenne Muscular DystrophyUnited States, Australia, Canada, United Kingdom, Japan, Bulgaria, Italy, Poland
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BioMarin PharmaceuticalNo longer availableDuchenne Muscular DystrophyUnited States, Germany, United Kingdom, Japan, Netherlands, France, Spain, Korea, Republic of, Italy, Belgium, Taiwan, Argentina, Australia, Bulgaria, Czechia, Israel, Norway, Poland, Russian Federation, Turkey
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AB2 Bio Ltd.CompletedNLRC4-MAS | XIAP DeficiencyUnited States, Canada, Germany
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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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University of FloridaEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsActive, not recruitingPyruvate Dehydrogenase Complex DeficiencyUnited States
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Stanford UniversityNational Heart, Lung, and Blood Institute (NHLBI); University of California... and other collaboratorsCompletedMuscular Dystrophy, DuchenneUnited States
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Baylor College of MedicineUniversity of California, San Diego; University of Alabama at Birmingham; Boston... and other collaboratorsCompletedPrader-Willi Syndrome | Rett Syndrome | Angelman Syndrome | Sleep ProblemsUnited States
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Masonic Cancer Center, University of MinnesotaRecruitingCerebral Adrenoleukodystrophy | Adrenoleukodystrophy | ALD (Adrenoleukodystrophy)United 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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Hugo W. Moser Research Institute at Kennedy Krieger...TerminatedAdrenoleukodystrophyUnited States
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AB2 Bio Ltd.Active, not recruitingNLRC4-MAS | XIAP DeficiencyUnited States, Canada, Germany
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Swedish Orphan BiovitrumIQVIA Pvt. LtdTerminatedHemophilia A | Hemophilia BBulgaria, Croatia, Czechia, France, Hungary, Ireland, Italy, Romania, Slovenia, Spain, United Kingdom
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Bio Products LaboratoryCompletedPrimary Immunodeficiency | Wiskott-Aldrich Syndrome | Hypogammaglobulinemia | Common Variable Hypogammaglobulinemia | X-linked Hypogammaglobulinemia | Immunodeficiency With Hyper-IgMUnited States
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rEVO BiologicsLaboratoire français de Fractionnement et de BiotechnologiesCompletedHemophilia A With Inhibitors | Hemophilia B With InhibitorsUnited States, Bulgaria, Poland, Russian Federation, Belarus, Georgia, Israel, Romania, Ukraine, United Kingdom
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsUnited States, Denmark, France, United Kingdom
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Mayo ClinicCompleted
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Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-Linked HypophosphatemiaUnited States
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ItalfarmacoCromsourceEnrolling by invitationDuchenne Muscular DystrophySpain, United States, Canada, Belgium, France, Israel, Italy, Netherlands, Serbia, Germany, United Kingdom
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Sarepta Therapeutics, Inc.Active, not recruitingMuscular Dystrophy, DuchenneUnited States, Korea, Republic of, India, Slovenia, Turkey, United Kingdom, Germany, Switzerland, Spain, Czechia, France, Serbia, Taiwan, Netherlands, Mexico, Colombia, Denmark, New Zealand, Norway, Greece, Hungary, Ireland, Italy, Jo... and more
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Genzyme, a Sanofi CompanyCompletedHemophilia A | Hemophilia BUnited States, Australia, Canada, China, France, Korea, Republic of, Malaysia, Turkey, Ukraine, United Kingdom, Germany, India, Italy, Japan, South Africa, Spain, Taiwan
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Baxalta now part of ShireCompletedHemophilia BTaiwan, Colombia, Argentina, Brazil, Bulgaria, Chile, Czechia, India, Ireland, Italy, Japan, Poland, Romania, Russian Federation, Sweden, Ukraine, United Kingdom
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Cooperative International Neuromuscular Research...CompletedMuscular Dystrophy, DuchenneUnited States
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Catabasis PharmaceuticalsCompletedMuscular Dystrophy, DuchenneUnited States, Canada, Australia, United Kingdom, Germany, Ireland, Israel, Sweden
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Bioverativ, a Sanofi companySwedish Orphan BiovitrumCompletedHemophilia BUnited States, Italy, Ireland, New Zealand, United Kingdom, Australia, Netherlands, Denmark, Poland, Sweden, France
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Amicus TherapeuticsActive, not recruitingFabry DiseaseUnited States, United Kingdom
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Kyowa Kirin, Inc.CompletedX-Linked HypophosphatemiaUnited States
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ApcinteX LtdCentessa Pharmaceuticals plcRecruitingHemophilia A | Hemophilia BUnited States, Italy, Spain, Taiwan, Australia, Belgium, United Kingdom, France, Germany, India, South Africa, Poland, Canada, Armenia, Turkey
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PfizerActive, not recruitingDuchenne Muscular DystrophyUnited States, Spain, Belgium, Korea, Republic of, Taiwan, Canada, Australia, Germany, Japan, France, Italy, United Kingdom, Russian Federation, Israel, Switzerland
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Genzyme, a Sanofi CompanyCompletedHemophilia A | Hemophilia BUnited States, Australia, China, Denmark, France, Israel, Japan, Korea, Republic of, Malaysia, Turkey, Ukraine, United Kingdom, Germany, India, South Africa, Spain, Taiwan, Hungary, Italy
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PTC TherapeuticsCooperative International Neuromuscular Research Group; The John Walton Muscular...Active, not recruitingMuscular Dystrophy, DuchenneGermany, France, United Kingdom, Brazil, Italy, Austria, Sweden, Czechia, Greece, Hungary, Israel, Latvia, Norway, Portugal, Romania, Slovenia
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Kevin FlaniganMuscular Dystrophy AssociationTerminated
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Genzyme, a Sanofi CompanyCompletedFabry DiseaseUnited States, France, Poland, United Kingdom