- ICH GCP
- US Clinical Trials Registry
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Search clinical trials for: x linked
Total 30497 results
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Sangamo TherapeuticsEnrolling by invitationFabry Disease | Fabry Disease, Cardiac VariantUnited States, Australia, United Kingdom
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Genzyme, a Sanofi CompanyTerminatedFabry Disease | Alpha Galactosidase A DeficiencyUnited States, Austria, United Kingdom
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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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ACADIA Pharmaceuticals Inc.CompletedRett SyndromeUnited States
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4D Molecular TherapeuticsActive, not recruiting
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4D Molecular TherapeuticsActive, not recruitingFabry DiseaseUnited States
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TakedaAvailableHunter SyndromeUnited States, Australia, Mexico, Spain, United Kingdom
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PfizerActive, not recruitingMuscular Dystrophy, DuchenneUnited States, Australia
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Amicus TherapeuticsEngage Health Inc.CompletedFabry DiseaseUnited States
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia BUnited States, Canada, United Kingdom, France, Malaysia, Brazil, Croatia, Germany, Italy, Japan, Taiwan
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Roswell Park Cancer InstituteActive, not recruitingAcute Myeloid Leukemia | Polycythemia Vera | Myelofibrosis | Chronic Myelomonocytic Leukemia | Waldenstrom Macroglobulinemia | Acute Lymphoblastic Leukemia | Chronic Lymphocytic Leukemia | Secondary Acute Myeloid Leukemia | Sickle Cell Disease | Myelodysplastic Syndrome | Plasma Cell Myeloma | Chronic Granulomatous... and other conditionsUnited States
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PTC TherapeuticsCompletedNonsene Mutation Duchenne Muscular DystrophyUnited States
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PfizerActive, not recruitingHemophilia A | Hemophilia BUnited States, Spain, Canada, China, Korea, Republic of, Hong Kong, Serbia, Taiwan, Turkey, France, Oman, Saudi Arabia, Japan, India, South Africa, Mexico, Croatia, Italy, Bulgaria, Russian Federation
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Novo Nordisk A/SEnrolling by invitationHaemophilia A | Haemophilia BJapan
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National Taiwan University HospitalCompletedNeurologic Disorder | Rett Syndrome
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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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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia BSpain, United Kingdom, Australia, Taiwan, France, Malaysia, United States, Austria, Canada, Israel, Japan, Thailand, Algeria, Argentina
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Novo Nordisk A/SActive, not recruitingHaemophilia A With Inhibitors | Haemophilia B With InhibitorsSpain, Korea, Republic of, India, Australia, United Kingdom, Russian Federation, Poland, Ukraine, Thailand, Mexico, United States, Turkey, Japan, South Africa, Serbia, France, Croatia, Denmark, Algeria, Bulgaria, Malaysia, Portugal, Slovaki... and more
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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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Beacon TherapeuticsEnrolling by invitationX-Linked Retinitis PigmentosaUnited States
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JCR Pharmaceuticals Co., Ltd.Active, not recruitingMucopolysaccharidosis IIJapan
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Bio Sidus SACompleted
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The Hospital for Sick ChildrenCompletedX-linked Lymphoproliferative Syndrome Type 2 (XLP-2)Canada
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Mitsubishi Tanabe Pharma America Inc.Active, not recruitingEPP | XLPUnited States, Germany, Japan, Spain, Italy, Sweden, Australia, Canada, Norway, United Kingdom
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Leipzig University Medical CenterEuropean Leukodystrophy AssociationCompletedX-linked AdrenoleukodystrophyGermany
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Caroline Michaela KistorpRigshospitalet, Denmark; Amicus TherapeuticsActive, not recruiting
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Sarepta Therapeutics, Inc.Hansa Biopharma ABActive, not recruitingDuchenne Muscular DystrophySpain
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FibroGenTerminatedDuchenne Muscular DystrophyUnited States, Belgium, Israel, Spain, Canada, Australia, Switzerland, United Kingdom, France, China, Netherlands, Italy, Austria, Czechia
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Gaziosmanpasa Research and Education HospitalCompletedDuchenne or Becker Muscular DystrophyTurkey
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Andrea GropmanChildren's National Research InstituteCompletedOrnithine Transcarbamylase Deficiency
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Daiichi Sankyo Co., Ltd.Orphan Disease Treatment Institute Co., Ltd.Completed
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Swedish Orphan BiovitrumCompletedHaemophilia A | Haemophilia BSweden, Finland, Denmark
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SanofiCompletedFabry DiseaseUnited Arab Emirates
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Sahra ŞirvanEnrolling by invitationDuchenne Muscular DystrophyTurkey
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Wave Life Sciences Ltd.Active, not recruitingDuchenne Muscular DystrophyJordan, United Kingdom
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Hacettepe UniversityCompleted
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Sheffield Children's NHS Foundation TrustSheffield Hallam University; National Institute for Health Research, United... and other collaboratorsActive, not recruiting
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National Human Genome Research Institute (NHGRI)Active, not recruitingWiskott- Aldrich Syndrome | ADA Deficient SCIDUnited States
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Spark TherapeuticsCompletedHematologic Diseases | Blood Coagulation Disorders, Inherited | Coagulation Protein Disorders | Hemorrhagic Disorders | Genetic Diseases, Inborn | Genetic Diseases, X-Linked | Gene Therapy | Blood Coagulation Disorder | Gene Transfer | Adeno-Associated Virus (AAV) | Factor VIII (FVIII) | Factor VIII (FVIII)... and other conditionsUnited States
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BiogenCompletedChoroideremiaUnited States, Germany, France
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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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Masonic Cancer Center, University of MinnesotaTerminatedSickle Cell Disease | Thalassemia | Cerebral Adrenoleukodystrophy | Inherited Metabolic Disorders | High Risk Hematologic DisordersUnited States
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Columbia UniversityMuscular Dystrophy Association; Stevens Institute of TechnologyCompletedDuchenne Muscular Dystrophy | Spinal Muscular Atrophy Type 3United 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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Avidity Biosciences, Inc.Enrolling by invitationDuchenne Muscular Dystrophy | DMD | Exon 44 | DuchenneUnited States
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Boston Children's HospitalMassachusetts General Hospital; Mayo Clinic; Karolinska University Hospital; Baylor... and other collaboratorsActive, not recruitingAplastic Anemia | Dyskeratosis Congenita | Hoyeraal Hreidarsson Syndrome | Revesz SyndromeUnited States, Norway, Sweden
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University of PittsburghRespirtech, Inc.CompletedDuchenne Muscular DystrophyUnited States
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Novo Nordisk A/SEnrolling by invitationHaemophilia BCanada, United Kingdom, Czechia, Germany, Greece, Austria, Belgium, Croatia, Denmark, Finland, Norway, Portugal