Search clinical trials for: x linked
Total 30233 results
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University of California, San FranciscoUniversity of Washington; California Institute of TechnologyRecruiting
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Genzyme, a Sanofi CompanyRecruitingFabry DiseaseCroatia, United States, Argentina, Australia, Belgium, Bulgaria, Canada, Germany, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Malaysia, Philippines, Poland, Romania, Singapore, Slovakia, Taiwan, Thailand, United Kingdom, Vietn... and more
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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 Eye Institute (NEI)Recruiting
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Eunice Kennedy Shriver National Institute of Child...RecruitingLysosomal Storage Disease | Cholesterol MetabolismUnited States
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National Cancer Institute (NCI)RecruitingFanconi Anemia | Dyskeratosis Congenita | Diamond Blackfan Anemia | Shwachman Diamond Syndrome | Inherited Bone Marrow Failure Syndrome, Aplastic AnemiaUnited States
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National Institute of Allergy and Infectious Diseases...RecruitingGranuloma | Granulomatous Disease, Chronic | Leukocyte Disease | Genetic Disease, X-Linked | Genetic Disease, InbornUnited States
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National Institute of Allergy and Infectious Diseases...Enrolling by invitationChronic Granulomatous Disease | p47 | Autosomal RecessiveUnited States
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Beacon TherapeuticsActive, not recruitingX-Linked Retinitis PigmentosaUnited States
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CSL BehringActive, not recruitingHemophilia BUnited States, Belgium, Netherlands, Denmark, Germany, Ireland, Sweden, United Kingdom
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Eunice Kennedy Shriver National Institute of Child...Completed
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National Cancer Institute (NCI)SuspendedHematopoietic Stem Cell Transplantation | ImmunodeficiencyUnited States
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Rett Syndrome Research TrustVanderbilt University Medical CenterCompleted
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National Institute of Mental Health (NIMH)CompletedHealthy Volunteers | Fragile X SyndromeUnited States
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University of California, DavisUniversity of Alberta; St. Justine's HospitalCompletedNeurobehavioral Manifestations | Genetic Diseases, X-Linked | Intellectual Disability | Fragile X Syndrome | Sex Chromosome Disorders | Fragile X Mental Retardation Syndrome | Trinucleotide Repeat Expansion | Fra(X) Syndrome | FXS | Mental Retardation, X LinkedUnited States, Canada
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National Institute of Allergy and Infectious Diseases...Active, not recruitingChronic Granulomatous Disease TransplantUnited States
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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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CSL BehringActive, not recruiting
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Medexus Pharma, Inc.CompletedHemophilia BTurkey, Brazil, Georgia, Moldova, Republic of, South Africa, Ukraine
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Sarepta Therapeutics, Inc.Hoffmann-La RocheEnrolling by invitationDuchenne Muscular DystrophyUnited States
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National Heart, Lung, and Blood Institute (NHLBI)CompletedCystic Fibrosis | Chronic Granulomatous Disease | Primary Ciliary Dyskinesia | Pseudohypoaldosteronism | Nontuberculous Mycobacterial InfectionUnited States
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Lokman Hekim ÜniversitesiCompletedParents | Duchenne Muscular Dystrophy | Psychoeducation | Psychological WellnessTurkey
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Edgewise Therapeutics, Inc.Medpace, Inc.Enrolling by invitationBecker Muscular DystrophyUnited States, Netherlands
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University of MalagaCompletedMuscular Dystrophy, Duchenne and Becker TypesSpain
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University Hospital Birmingham NHS Foundation TrustNorthern Care Alliance NHS Foundation Trust; Cambridge University Hospitals... and other collaboratorsActive, not recruitingArrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease (RaILRoAD)Fabry DiseaseUnited Kingdom, Australia
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4D Molecular TherapeuticsActive, not recruitingChoroideremiaUnited States
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Sangamo TherapeuticsActive, not recruitingFabry DiseaseUnited States, United Kingdom, Taiwan, Australia, Germany, Italy, Canada
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Nova Mentis Life Science CorpKGK Science Inc.SuspendedCognitive Dysfunction | Fragile X Syndrome | BehaviorCanada
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ShireActive, not recruiting
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ShireCompletedHunter SyndromeUnited States, United Kingdom, Canada
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ShireCompletedHunter SyndromeUnited States, Spain, Canada, United Kingdom, Mexico, Australia, France
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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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Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-linked HypophosphatemiaNetherlands, United States, United Kingdom, France
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Zynerba Pharmaceuticals, Inc.Enrolling by invitationFragile X SyndromeUnited States, United Kingdom, New Zealand, Australia
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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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Kyowa Kirin Pharmaceutical Development LtdCompletedX-linked Hypophosphatemia (XLH)Sweden, Spain, United Kingdom, Italy, Austria, France
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Sangamo TherapeuticsActive, not recruitingHemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis IIUnited States
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National Institute of Allergy and Infectious Diseases...Enrolling by invitationSafety and Efficacy of Tofacitinib for Chronic Granulomatous Disease With Inflammatory ComplicationsChronic Granulomatous Disease | Inflammatory Gastrointestinal Disease | Inflammatory Skin Disease | Inflammatory Lung DiseaseUnited States
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Santhera PharmaceuticalsSanthera PharmaceuticalsActive, not recruitingDuchenne Muscular DystrophyCanada
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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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RTI InternationalUniversity of North Carolina, Chapel HillEnrolling by invitationTuberous Sclerosis | Down Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Rett Syndrome | Turner Syndrome | Williams Syndrome | Angelman Syndrome | Chromosome 22q11.2 Deletion Syndrome | Klinefelter Syndrome | Phelan-McDermid Syndrome | Dup15Q Syndrome | Smith Magenis SyndromeUnited States
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CSL BehringEnrolling by invitation
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University of NebraskaUniversity of Colorado, Denver; University of Pittsburgh; Children's Hospital...Completed
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Vastra Gotaland RegionKarolinska University Hospital; Göteborg University; Uppsala University HospitalCompleted
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Ultragenyx Pharmaceutical IncActive, not recruitingOrnithine Transcarbamylase (OTC) DeficiencyCanada, United States, France, Spain, United Kingdom
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VegaVect, Inc.National Eye Institute (NEI)Active, not recruiting
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Novo Nordisk A/SActive, not recruiting
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Denali Therapeutics Inc.Active, not recruitingMucopolysaccharidosis IIUnited States, Netherlands, Canada, United Kingdom