Clinical Trials on Genetic Diseases, X-Linked in United States
Total 7980 results
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Bio Products LaboratoryCompletedHaemophilia BUnited Kingdom, Poland
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Baxalta now part of ShireCompletedHemophilia BPoland, United Kingdom, Spain, Bulgaria, Romania, Japan, Russian Federation, Brazil, Chile, Sweden, Colombia, Ukraine, Argentina, Czechia
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BioMarin PharmaceuticalTerminatedDuchenne Muscular DystrophyUnited States, Netherlands, Belgium, Germany, Argentina, Brazil, France, Italy, Sweden, Turkey
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PTC TherapeuticsCompletedNervous System Diseases | Genetic Diseases, Inborn | Genetic Diseases, X-Linked | Musculoskeletal Diseases | Muscular Diseases | Neuromuscular Diseases | Muscular Dystrophies | Muscular Disorders, Atrophic | Muscular Dystrophy, DuchenneUnited States, Spain, Korea, Republic of, Belgium, France, Canada, Australia, United Kingdom, Israel, Italy, Brazil, Chile, Czechia, Sweden, Switzerland, Turkey, Germany, Poland
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Novo Nordisk A/SCompletedHaemophilia A | Haemophilia BUnited States
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Santhera PharmaceuticalsCompletedMuscular Dystrophy, Duchenne | Ambulatory CareUnited States, Spain, Austria, Belgium, France, Germany, Italy, Netherlands, Sweden, Switzerland
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PfizerRecruitingHemophilia A | Hemophilia BHong Kong, Spain, Serbia, Turkey, United States, China, Mexico, South Africa, India, Japan, Taiwan, Croatia, Korea, Republic of, Oman, Italy, Canada, France
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Baxalta now part of ShireCompletedHemophilia A | Hemophilia B | Hemophilia A or B With InhibitorsPoland, United States, Bulgaria, Romania, Japan, Russian Federation, New Zealand, Croatia, Ukraine, Brazil
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PTC TherapeuticsGenzyme, a Sanofi CompanyTerminatedDuchenne Muscular Dystrophy | Becker Muscular DystrophyUnited States, United Kingdom
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Sarepta Therapeutics, Inc.Active, not recruitingDuchenne Muscular DystrophyUnited States, Korea, Republic of, Italy, United Kingdom, Canada, Poland, Spain, France, Germany, Belgium, Australia, Czechia, Serbia, Greece, Argentina, Bulgaria, Denmark, Hungary, India, Ireland, Israel, Mexico, Russian Federation, ...
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Laboratoire français de Fractionnement et de BiotechnologiesLFB USA, Inc.CompletedA Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa (PERSEPT2)Hemophilia A With Inhibitors | Hemophilia B With InhibitorsBulgaria, Ukraine, Czechia, United States, Georgia, South Africa
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Arcturus Therapeutics, Inc.Active, not recruitingOrnithine Transcarbamylase DeficiencyUnited States
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Sangamo TherapeuticsActive, not recruitingHemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis IIUnited States
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Novo Nordisk A/SEnrolling by invitationHaemophilia BCanada, United Kingdom, Czechia, Germany, Greece, Austria, Belgium, Croatia, Denmark, Finland, Norway, Portugal
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Northwell HealthNational Cancer Institute (NCI); University of California, San DiegoCompletedHemophilia A | Hemophilia B | HemarthrosesUnited States
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PfizerActive, not recruitingHemophilia A | Hemophilia BUnited States, Canada, Spain, Australia, Korea, Republic of, Taiwan, Brazil, Italy, Japan, Turkey, Germany, United Kingdom, Saudi Arabia, France, Sweden, Belgium, Greece, Israel
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Cooperative International Neuromuscular Research...United States Department of Defense; National Institutes of Health (NIH); U.S... and other collaboratorsUnknownDuchenne Muscular DystrophyUnited States, Canada, Australia, India, Israel, Argentina, Italy, Sweden
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Dyne TherapeuticsRecruitingDuchenne Muscular Dystrophy (DMD)United States, Belgium, Canada, United Kingdom, Australia, Spain, Italy
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CSL BehringActive, not recruitingHemophilia BUnited States, Belgium, Netherlands, Denmark, Germany, Ireland, Sweden, United Kingdom
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Nationwide Children's HospitalBoston Children's Hospital; University of California, Davis; University of Minnesota and other collaboratorsCompletedCardiomyopathy | Duchenne Muscular Dystrophy (DMD)United States
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ApcinteX LtdCentessa Pharmaceuticals plcRecruitingHemophilia A | Hemophilia BSpain, Taiwan, United Kingdom, Italy, Germany, United States, South Africa, Australia, Poland, Armenia, France, India, Canada, Brazil, Turkey, Egypt
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Ultragenyx Pharmaceutical IncTerminatedCreatine Deficiency, X-linkedUnited States, Canada
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Cooperative International Neuromuscular Research...National Institutes of Health (NIH)CompletedDuchenne Muscular Dystrophy | Becker Muscular Dystrophy | Limb Girdle Muscular DystrophyUnited States
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Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-Linked HypophosphatemiaKorea, Republic of, Canada, United States, Australia, Japan, United Kingdom, Sweden
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University College, LondonRecruiting
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Gillette Children's Specialty HealthcareCompletedCerebral Palsy | Muscular Dystrophy | Osteopenia | Rett Syndrome | Spinal Muscular Atrophy | Spina BifidaUnited States
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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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Stephen G. Kaler, MDRecruitingMenkes Disease | Occipital Horn SyndromeUnited States
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Santhera PharmaceuticalsTerminatedDuchenne Muscular Dystrophy (DMD)United States, Spain, Netherlands, Sweden, Germany, France, Belgium, United Kingdom, Italy, Ireland, Switzerland, Austria, Bulgaria, Hungary, Israel
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Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-Linked HypophosphatemiaUnited States
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Great Ormond Street Hospital for Children NHS Foundation...National Institute for Health Research, United Kingdom; The Freya FoundationRecruitingPyruvate Dehydrogenase Complex Deficiency | Pyruvate Dehydrogenase E1 Alpha Deficiency | Pyruvate Dehydrogenase E1-Beta Deficiency | Pyruvate Dehydrogenase E2 Deficiency | Pyruvate Dehydrogenase Phosphatase DeficiencyUnited Kingdom
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Great Ormond Street Hospital for Children NHS Foundation...Catholic University of the Sacred Heart; Università degli Studi di Ferrara; Universidad... and other collaboratorsRecruitingDuchenne Muscular Dystrophy | Becker Muscular DystrophyDenmark, France, Italy, Netherlands, Spain, United Kingdom
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Astellas Gene TherapiesActive, not recruitingX-Linked Myotubular MyopathyUnited States, Canada, France, Germany
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Bio Products LaboratoryCompletedCommon Variable Immunodeficiency | Wiskott-Aldrich Syndrome | X-linked Agammaglobulinemia | Primary Immune Deficiency Disorders | Hyper-IgM SyndromeUnited States, Chile, Israel
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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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University College, LondonRecruitingOrnithine Transcarbamylase DeficiencyUnited Kingdom
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Kyowa Kirin, Inc.CompletedX-Linked HypophosphatemiaUnited 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 Allergy and Infectious Diseases...WithdrawnChronic Granulomatous Disease | Inflammatory Bowel DiseaseUnited States
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GlaxoSmithKlineTerminatedMuscular DystrophiesFrance, Bulgaria, Denmark, Germany, Belgium, Canada, Spain, Turkey, United Kingdom, Korea, Republic of, Brazil, Netherlands, Italy, Japan, Czech Republic, Chile, Israel, Hungary, Russian Federation, Australia, Poland, Norway, Argentina and more
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Weill Medical College of Cornell UniversityNovartis PharmaceuticalsTerminatedBleeding | Thrombocytopenia | Wiskott-Aldrich SyndromeUnited States
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National Institute of Allergy and Infectious Diseases...CompletedGranulomatous Disease, Chronic | Infection | Job's SyndromeUnited States
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Cooperative International Neuromuscular Research...United States Department of DefenseCompletedDuchenne Muscular Dystrophy | Becker Muscular Dystrophy | Limb Girdle Muscular DystrophyUnited States, Japan, Canada
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Wyeth is now a wholly owned subsidiary of PfizerCompletedHemophilia BBelgium, Spain, Italy, Austria, United Kingdom, Netherlands, Sweden, Germany, Portugal, France
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PfizerRecruitingHemophilia A | Hemophilia BDenmark, Taiwan, Canada, Turkey, Spain, China, United Kingdom, South Africa, Italy, Saudi Arabia, Israel, Korea, Republic of, Austria, Germany, Japan, Australia, India, Slovakia, France, United States, Brazil, Argentina
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Massachusetts General HospitalUniversity of Pittsburgh; Boston Children's Hospital; Brigham and Women's Hospital and other collaboratorsRecruitingDuchenne Muscular Dystrophy | Becker Muscular Dystrophy | Myotonic Dystrophy | Facioscapulohumeral Muscular DystrophyUnited States
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PfizerActive, not recruitingHemophilia BGermany, United Kingdom, Canada, Australia, Taiwan, Japan, France, Turkey, Korea, Republic of, United States, Brazil, Greece, Saudi Arabia, Sweden
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Swedish Orphan BiovitrumCompletedHaemophilia A | Haemophilia BFrance, Germany, Italy, Spain, Sweden, Belgium, United Kingdom
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PTC TherapeuticsGenzyme, a Sanofi CompanyTerminatedHemophilia A | Hemophilia BUnited States, Canada, France, Italy
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Sarepta Therapeutics, Inc.Completed