Clinical Trials on Genetic Diseases, X-Linked in South Africa
Total 1107 results
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Catalyst BiosciencesCompletedHemophilia BSouth Africa
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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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BayerCompletedHemophilia A | Hemophilia BSouth Africa, Poland, United Kingdom
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Anavex Life Sciences Corp.Completed
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Mitsubishi Tanabe Pharma America Inc.RecruitingErythropoietic Protoporphyria (EPP) | X-Linked Protoporphyria (XLP)United States, Spain, France, Japan, United Kingdom, Italy, Australia, Poland, Bulgaria, Czechia, Netherlands
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Zynerba Pharmaceuticals, Inc.RecruitingFragile X SyndromeUnited States, Australia, United Kingdom, Ireland
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Zynerba Pharmaceuticals, Inc.CompletedFragile X SyndromeUnited States, New Zealand, Australia
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Anavex Life Sciences Corp.International Rett Syndrome Foundation Rettsyndrome.orgCompleted
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Anavex Life Sciences Corp.Anavex Australia Pty Ltd.; Anavex Germany GmbHCompletedRett SyndromeCanada, Australia, United Kingdom
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Zynerba Pharmaceuticals, Inc.Enrolling by invitationFragile X SyndromeUnited States, United Kingdom, New Zealand, Australia
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Novartis PharmaceuticalsCompletedFragile X SyndromeUnited States, Switzerland, Germany, Australia, Canada, Denmark, France, Italy, Spain, United Kingdom
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Catalyst BiosciencesCompletedHemophilia A With Inhibitor | Hemophilia B With InhibitorArmenia, Georgia, South Africa, Poland, Russian Federation
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Medexus Pharma, Inc.CompletedHemophilia BTurkey, Brazil, Georgia, Moldova, Republic of, South Africa, Ukraine
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Novo Nordisk A/SCompletedEpidemiological Study on Haemophilia Care and Orthopaedic Status in Developing Countries (HAEMOcare)Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors | Haemophilia A | Haemophilia BIndia, Morocco, South Africa, Oman
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Neuren Pharmaceuticals Limitedrettsyndrome.orgCompleted
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Jazz PharmaceuticalsGW PharmaceuticalsTerminatedRett Syndrome | RTTUnited States, Canada, Italy, Spain, United Kingdom, Australia
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Tetra Discovery PartnersRecruitingFragile X SyndromeUnited States
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Tetra Discovery PartnersEnrolling by invitationFragile X SyndromeUnited States
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Hoffmann-La RocheCompleted
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Neuren Pharmaceuticals LimitedCompleted
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ACADIA Pharmaceuticals Inc.CompletedRett SyndromeUnited States
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ACADIA Pharmaceuticals Inc.Completed
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Seaside Therapeutics, Inc.Completed
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Novartis PharmaceuticalsTerminatedFragile X SyndromeGermany, United States, Switzerland, Australia, Canada, Denmark, France, Italy, United Kingdom, Spain
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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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University of Alabama at BirminghamEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompletedCDKL5 | Rett Syndrome | MECP2 Duplication | FOXG1 DisordersUnited States
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University of Alabama at BirminghamUniversity of Colorado, Denver; Eunice Kennedy Shriver National Institute of... and other collaboratorsCompletedRett Syndrome | MECP2 Duplication Disorder | Rett-related DisorderUnited States
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ACADIA Pharmaceuticals Inc.CompletedRett SyndromeUnited States
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International Rett Syndrome FoundationBaylor College of Medicine; Children's Hospital of Philadelphia; University of... and other collaboratorsRecruitingNervous System Diseases | Neurologic Manifestations | Neurobehavioral Manifestations | Genetic Diseases, X-Linked | Intellectual Disability | Neurodevelopmental Disorders | Neurologic Disorder | Rett Syndrome | Genetic Disease | Rett Syndrome, AtypicalUnited States
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Kiora Pharmaceuticals, Inc.RecruitingRetinitis Pigmentosa | ChoroideremiaAustralia
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Novartis PharmaceuticalsTerminatedFragile X SyndromeUnited States, Belgium, Australia, Israel, Switzerland, Germany, Denmark, Italy, Netherlands, Spain, Sweden, United Kingdom, France
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Jazz PharmaceuticalsTerminatedRett Syndrome | RTTUnited States, Spain, Italy, United Kingdom
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Novartis PharmaceuticalsCompletedFragile X SyndromeUnited States, Belgium, Australia, Israel, Switzerland, Germany, France, Canada, Denmark, Indonesia, Italy, Netherlands, Spain, Sweden, Turkey, United Kingdom
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University of Alabama at BirminghamEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompletedRett Syndrome | CDKL5 Disorder | MECP2 Duplication dIsorder | FOXG1 SyndromeUnited States
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CSL BehringTerminatedHemophilia A With Inhibitors | Hemophilia B With InhibitorsGeorgia, Italy, Malaysia, Russian Federation, South Africa, Spain, Thailand, Ukraine, United Kingdom
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Bioverativ Therapeutics Inc.Swedish Orphan BiovitrumCompletedHemophilia BUnited States, United Kingdom, South Africa, Netherlands, Ireland, Australia
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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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Denali Therapeutics Inc.CompletedMucopolysaccharidosis IIUnited Kingdom, United States, Italy, Netherlands
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Freeline TherapeuticsTerminatedHemophilia BUnited Kingdom
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Denali Therapeutics Inc.Active, not recruitingMucopolysaccharidosis IIUnited States, Netherlands, Canada, United Kingdom
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Spark TherapeuticsChildren's Hospital of Philadelphia; University of Pittsburgh; St. James's Hospital... and other collaboratorsTerminated
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Baylor College of MedicineUniversity of California, San Diego; University of Alabama at Birmingham; Boston... and other collaboratorsCompletedPrader-Willi Syndrome | Rett Syndrome | Angelman Syndrome | Sleep ProblemsUnited States
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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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ApcinteX LtdCentessa Pharmaceuticals plcRecruitingHemophilia A | Hemophilia BUnited States, Italy, Spain, Taiwan, Australia, Belgium, United Kingdom, France, Germany, India, South Africa, Poland, Canada, Armenia, Turkey
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ApcinteX LtdCentessa Pharmaceuticals plcRecruitingHemophilia A | Hemophilia BSpain, Taiwan, United Kingdom, Italy, Germany, United States, South Africa, Australia, Egypt, Poland, Armenia, France, India, Canada, Brazil, Turkey
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CSL BehringCompletedHemophilia BUnited States, Australia, Japan, France, Philippines, Spain, Czechia, Israel, Canada, Germany, Austria, Bulgaria, Italy, Malaysia, South Africa
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Novo Nordisk A/SCompletedHealthy | Congenital Bleeding Disorder | Haemophilia A | Haemophilia BAustria, Sweden, Spain, Switzerland, United Kingdom, Germany, Malaysia, Denmark, South Africa, Thailand, Italy
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Genzyme, a Sanofi CompanyCompletedHemophilia A | Hemophilia BUnited States, Australia, Canada, China, France, Korea, Republic of, Malaysia, Turkey, Ukraine, United Kingdom, Germany, India, Italy, Japan, South Africa, Spain, Taiwan
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PfizerActive, not recruitingMuscular Dystrophy, DuchenneUnited States, Australia