Search clinical trials for: Genetic Diseases, X-Linked
Total 18734 results
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National Institute of Allergy and Infectious Diseases...RecruitingChronic Granulomatous Disease (CGD) | X-Linked Chronic Granulomatous DiseaseUnited States
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iECURE, Inc.RecruitingUrea Cycle Disorders, Inborn | Ornithine Transcarbamylase Deficiency | Ornithine Transcarbamylase Deficiency Disease | Ornithine Carbamoyltransferase Deficiency (Disorder)United Kingdom
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Hospices Civils de LyonNot yet recruitingX-Linked Hypophosphatemic Rickets
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Assistance Publique - Hôpitaux de ParisNot yet recruitingHemophilia A | Hemophilia B | Cystic Fibrosis | Sickle Cell Disease | Muscular Dystrophy, Duchenne | Fragile X Syndrome | Huntington Disease | Myotonic Dystrophy | Autosomal Recessive Polycystic Kidney Disease | Neurofibromatosis-Noonan Syndrome | Muscular Dystrophy, Becker | Invasive PreNatal Diagnosis in a... and other conditionsFrance
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Mitsubishi Tanabe Pharma America Inc.RecruitingErythropoietic Protoporphyria (EPP) | X-Linked Protoporphyria (XLP)Spain, United States, France, Japan, United Kingdom, Italy, Australia, Poland, Bulgaria, Czechia, Netherlands
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Janssen Pharmaceutical K.K.RecruitingX-Linked Retinitis PigmentosaJapan
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Hereditary Neuropathy FoundationRecruitingCharcot-Marie-Tooth Disease | Charcot-Marie-Tooth Disease, Type IA | Charcot-Marie-Tooth Disease Type 2A | Charcot-Marie-Tooth | Charcot-Marie-Tooth Disease, Type IB | Charcot-Marie-Tooth Disease Type 2 | Charcot-Marie-Tooth Disease, Type 2C | Charcot-Marie-Tooth Disease Type 2A2B | Charcot-Marie-Tooth... and other conditionsUnited States
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Frontera TherapeuticsPeking Union Medical College Hospital; Eye & ENT Hospital of Fudan University; Henan Provincial People's HospitalRecruitingX-Linked Retinitis PigmentosaChina
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EspeRare FoundationIqvia Pty Ltd; Pierre Fabre MedicamentRecruitingX-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)United States, Spain, France, Germany, Italy, United Kingdom
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Beacon TherapeuticsRecruitingX-Linked Retinitis PigmentosaUnited States
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Children's Hospital of Chongqing Medical UniversityRecruiting
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Great Ormond Street Hospital for Children NHS Foundation...RecruitingSevere Combined Immunodeficiency, X-LinkedUnited Kingdom
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Pitié-Salpêtrière HospitalRecruitingLaminopathies | EmerinopathiesFrance
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University of California, Los AngelesNational Heart, Lung, and Blood Institute (NHLBI); Boston Children's Hospital; California Institute for Regenerative Medicine (CIRM) and other collaboratorsRecruitingGranulomatous Disease, Chronic, X-linkedUnited States
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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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National Institute of Allergy and Infectious Diseases...RecruitingX-linked Severe Combined Immunodeficiency (XSCID)United States
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National Institute of Allergy and Infectious Diseases...RecruitingChronic Granulomatous Disease (CGD) | X-Linked Severe Combined Immune Deficiency (XSCID) | Leukocyte Adhesion Deficiency 1 (LAD) | Graft Versus Host Disease (cGvHD)United States
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National Human Genome Research Institute (NHGRI)Active, not recruitingWiskott- Aldrich Syndrome | ADA Deficient SCIDUnited States
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Janssen Research & Development, LLCJanssen Research & Development, LLCActive, not recruitingX-Linked Retinitis PigmentosaBelgium, Canada, United States, Israel, United Kingdom, Denmark, France, Italy, Netherlands, Switzerland, Spain
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Kyowa Kirin Pharmaceutical Development LtdCompletedX-Linked HypophosphatemiaFrance, United Kingdom, Spain, Germany, Netherlands
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Janssen Research & Development, LLCJanssen Research & Development, LLCActive, not recruitingX-Linked Retinitis PigmentosaUnited States, Canada, Israel, United Kingdom, Denmark, France, Belgium, Italy, Netherlands, Switzerland, Spain
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NightstaRx Ltd, a Biogen CompanyEnrolling by invitationChoroideremia | X-Linked Retinitis PigmentosaUnited States, United Kingdom, Netherlands, Germany, France, Brazil, Canada, Denmark, Finland
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Janssen Research & Development, LLCActive, not recruitingX-Linked Retinitis PigmentosaUnited States, United Kingdom
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Janssen Pharmaceutical K.K.CompletedX-Linked Retinitis PigmentosaJapan
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Beacon TherapeuticsActive, not recruitingX-Linked Retinitis PigmentosaUnited States
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Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-Linked HypophosphatemiaUnited States
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Kyowa Kirin, Inc.CompletedX-Linked HypophosphatemiaUnited States
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David WilliamsActive, not recruitingGene Therapy | Severe Combined Immunodeficiency, X LinkedUnited States, United Kingdom
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St. Jude Children's Research HospitalNational Heart, Lung, and Blood Institute (NHLBI); Assisi Foundation; California...SuspendedSevere Combined Immunodeficiency Disease, X-linkedUnited States
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Murdoch Childrens Research InstituteUniversity of Sydney; Sydney Children's Hospitals Network; Women's and Children... and other collaboratorsActive, not recruitingAutosomal Recessive Disorder | X-Linked Genetic DiseasesAustralia
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4D Molecular TherapeuticsActive, not recruiting
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4D Molecular TherapeuticsActive, not recruitingFabry DiseaseUnited States
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Beacon TherapeuticsActive, not recruitingX-Linked Retinitis PigmentosaUnited States
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RTI InternationalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationPrimary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditionsUnited States
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Beacon TherapeuticsEnrolling by invitationX-Linked Retinitis PigmentosaUnited States
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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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BiogenCompletedX-Linked Retinitis PigmentosaUnited States, United Kingdom
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4D Molecular TherapeuticsActive, not recruitingX-Linked Retinitis PigmentosaUnited States
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Kyowa Kirin Co., Ltd.Active, not recruiting
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Kyowa Kirin Pharmaceutical Development LtdCompletedX-Linked HypophosphataemiaUnited Kingdom
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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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Kyowa Kirin Pharmaceutical Development LtdActive, not recruitingX-Linked HypophosphatemiaSpain, Italy, United Kingdom, France, Belgium, Denmark, Germany, Hungary, Portugal, Norway, Switzerland, Israel, Netherlands, Czechia, Bulgaria, Sweden, Slovenia, Ireland, Latvia, Slovakia
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GenethonActive, not recruitingX-Linked Chronic Granulomatous DiseaseFrance
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GenethonActive, not recruitingX-Linked Chronic Granulomatous DiseaseUnited Kingdom
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NightstaRx Ltd, a Biogen CompanyCompletedX-Linked Retinitis PigmentosaUnited States, France, United Kingdom, Finland, Netherlands, Germany, Canada
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Applied Genetic Technologies CorpCompletedX-Linked Retinitis PigmentosaUnited States
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MeiraGTx UK II LtdSyne Qua Non Limited; Bionical EmasCompletedGene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)X-Linked Retinitis PigmentosaUnited Kingdom, United States
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University of the Philippines Manila - Philippine...AvailableX-Linked Dystonia ParkinsonismPhilippines
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Assistance Publique - Hôpitaux de ParisCompletedX-linked Severe Combined ImmunodeficiencyFrance
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University Hospital ErlangenPervormance GmbHCompletedShort Term Effects and Risks of Physical Exercise in Subjects With Hypohidrotic Ectodermal DysplasiaX-Linked Hypohidrotic Ectodermal Dysplasia | Exercise-induced Rise of Body TemperatureGermany