Clinical Trials on X-Linked+Combined+Immunodeficiency+Diseases
Total 4406 results
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Rocket Pharmaceuticals Inc.Active, not recruitingDanon DiseaseUnited States
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Karen Brorup Heje PedersenCompletedHealthy Subjects | Spinal and Bulbar Muscular AtrophyDenmark
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Ramathibodi HospitalAstraZenecaCompletedSpinobulbar Muscular Atrophy | Kennedy's DiseaseThailand
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Federal Research Institute of Pediatric Hematology...RecruitingWiskott-Aldrich SyndromeRussian Federation
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Weill Medical College of Cornell UniversityNovartis PharmaceuticalsTerminatedBleeding | Thrombocytopenia | Wiskott-Aldrich SyndromeUnited States
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Assistance Publique - Hôpitaux de ParisInstitut National de la Santé Et de la Recherche Médicale, France; Association...RecruitingAmyotrophic Lateral Sclerosis | Kennedy DiseaseFrance
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National Institute of Neurological Disorders and...CompletedSpinal and Bulbar Muscular Atrophy | Kennedy's DiseaseUnited States
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Janssen-Ortho Inc., CanadaCompletedAutistic Disorder | Developmental Disabilities | Rett Syndrome | Asperger Syndrome | DCild Development Disorders, Pervasive
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Rocket Pharmaceuticals Inc.RecruitingDanon DiseaseUnited States
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Denali Therapeutics Inc.CompletedMucopolysaccharidosis IIUnited Kingdom, United States, Italy, Netherlands
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JCR Pharmaceuticals Co., Ltd.Active, not recruitingMucopolysaccharidosis IIJapan
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ShireCompleted
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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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JCR Pharmaceuticals Co., Ltd.Completed
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JCR Pharmaceuticals Co., Ltd.Completed
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Sangamo TherapeuticsTerminatedMucopolysaccharidosis II | MPS IIUnited States
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REGENXBIO Inc.Withdrawn
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Green Cross CorporationCompletedMucopolysaccharidosis IIKorea, Republic of
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Eunice Kennedy Shriver National Institute of Child...University of MinnesotaCompleted
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Bionano GenomicsColumbia University; University of Iowa; Medical College of Wisconsin; Augusta... and other collaboratorsRecruitingIntellectual Disability | Autism Spectrum Disorder | Fragile X Syndrome | Developmental Disability | Congenital Anomaly | Facioscapulohumeral Muscular Dystrophy 1United States
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National Human Genome Research Institute (NHGRI)CompletedAutism | Asperger's Syndrome | Rett's DisorderUnited States
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University of Mississippi Medical CenterCompletedAutistic Disorder | Rett Syndrome | Asperger Syndrome | Pervasive Developmental Disorder | Childhood Disintegrative DisorderUnited States
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Janssen Pharmaceutical K.K.RecruitingX-Linked Retinitis PigmentosaJapan
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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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Janssen Pharmaceutical K.K.RecruitingX-Linked Retinitis PigmentosaJapan
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AO GENERIUMRecruitingMetabolic Diseases | Mucopolysaccharidosis Type IIRussian Federation
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JCR Pharmaceuticals Co., Ltd.RecruitingMucopolysaccharidosis IIUnited States, Germany, Spain, France, Brazil, Argentina, Turkey, United Kingdom, Poland, Italy
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Denali Therapeutics Inc.Active, not recruitingMucopolysaccharidosis IIUnited States, Netherlands, Canada, United Kingdom
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Denali Therapeutics Inc.Enrolling by invitationMucopolysaccharidosis IIUnited States, Canada, Belgium, Czechia, Netherlands
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JCR Pharmaceuticals Co., Ltd.Enrolling by invitationMucopolysaccharidosis IIUnited States
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JCR Pharmaceuticals Co., Ltd.Completed
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REGENXBIO Inc.CompletedMucopolysaccharidosis IIBrazil, United States, United Kingdom
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REGENXBIO Inc.Enrolling by invitation
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ArmaGen, IncCompletedMucopolysaccharidosis IIUnited States, Germany, Philippines
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TakedaRecruiting
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TakedaAvailableHunter SyndromeUnited States, Australia, Mexico, Spain, United Kingdom
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Green Cross CorporationCompleted
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OctapharmaCompletedPrimary ImmunodeficiencyUnited Kingdom, Australia, Germany, Italy
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NightstaRx Ltd, a Biogen CompanyCompletedChoroideremiaFinland, United States, Canada, Denmark, Netherlands, Germany, United Kingdom, Brazil, France
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Hacettepe UniversityCompletedChewing Problem | Swallowing DisorderTurkey
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Janssen Research & Development, LLCJanssen Research & Development, LLCActive, not recruitingX-Linked Retinitis PigmentosaUnited States, Canada, Israel, United Kingdom, Spain, Denmark, France, Belgium, Italy, Netherlands, Switzerland
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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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Applied Genetic Technologies CorpCompletedX-Linked Retinitis PigmentosaUnited States
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National Center for Research Resources (NCRR)University of California, San DiegoCompleted
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Beacon TherapeuticsActive, not recruitingX-Linked Retinitis PigmentosaUnited States
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Beacon TherapeuticsEnrolling by invitationX-Linked Retinitis PigmentosaUnited States
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Beacon TherapeuticsActive, not recruitingX-Linked Retinitis PigmentosaUnited States
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University of ManchesterManchester University NHS Foundation Trust; Great Ormond Street Hospital for... and other collaboratorsRecruiting
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GC Biopharma CorpRecruitingMucopolysaccharidosis II | Hunter SyndromeKorea, Republic of