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Clinical Trials on X-linked Adrenal Hypoplasia Congenita
Total 2203 results
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Massachusetts General HospitalEuropean Leukodystrophy AssociationRecruitingRestless Legs Syndrome | AdrenoleukodystrophyUnited States, Netherlands
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Minoryx Therapeutics, S.L.Active, not recruitingCerebral AdrenoleukodystrophySpain, Germany, France, Argentina
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University of the Philippines Manila - Philippine...AvailableX-Linked Dystonia ParkinsonismPhilippines
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Ultragenyx Pharmaceutical IncTerminatedCreatine Deficiency, X-linkedUnited States, Canada
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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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University Hospital ErlangenEdimer PharmaceuticalsCompletedHypohidrotic Ectodermal DysplasiaGermany
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GenethonActive, not recruitingX-Linked Chronic Granulomatous DiseaseFrance
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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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Rigshospitalet, DenmarkCompleted
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Ariel UniversityAssociazione Italiana Rett (AIRETT)Not yet recruitingRett SyndromeItaly
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Haukeland University HospitalKarolinska Institutet; University Hospitals Bristol and Weston NHS Foundation... and other collaboratorsCompletedCongenital Adrenal Hyperplasia | Acromegaly | Growth Hormone Deficiency | Cushing Syndrome | Primary Hyperaldosteronism | Adrenal InsufficiencyNorway, Sweden, Greece, United Kingdom
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University Hospital, MontpellierAgence de La BiomédecineActive, not recruitingFragile X Syndrome | Huntington Disease | Myotonic Dystrophy 1France
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GenethonActive, not recruitingX-Linked Chronic Granulomatous DiseaseUnited Kingdom
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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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IRCCS Fondazione Stella MarisRecruitingCreatine Deficiency, X-linked | Creatine Deficiency Syndrome Due to Gamt DeficiencyItaly
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The University of QueenslandRecruitingPulmonary Fibrosis | Dyskeratosis Congenita | Telomere Shortening | Telomere DiseaseAustralia
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Ariel UniversityASST Santi Paolo e CarloCompleted
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Icahn School of Medicine at Mount SinaiNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Rare... and other collaboratorsCompletedErythropoietic Protoporphyria | EPP | XLP | X-Linked Protoporphyria | XLPP | X-Linked Dominant Erythropoietic Protoporphyria | XLEPP | XLDPUnited States
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Jakub AntczakCompletedAdrenomyeloneuropathy | Hereditary Spastic ParaplegiaPoland
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Rare Thyroid Therapeutics International ABVitaccess LtdCompletedAllan-Herndon-Dudley Syndrome | Monocarboxylate Transporter 8 (MCT8) DeficiencyUnited Kingdom
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Great Ormond Street Hospital for Children NHS Foundation...WithdrawnGranulomatous Disease, Chronic, X-linked, VariantUnited Kingdom
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National Institute of Allergy and Infectious Diseases...CompletedImmunoproliferative DisorderUnited States
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Duke UniversityUniversity of FloridaCompleted
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Washington University School of MedicineCompletedBarth Syndrome
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Queen Fabiola Children's University HospitalUnknownCongenital Hypoplasia of Aortic ArchBelgium
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Baylor College of MedicineCompleted
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Bio Products LaboratoryCompletedCommon Variable Immunodeficiency | Primary Immune Deficiency Disorders | X-linked Agammaglobulinaemia | Hyperimmunoglobulin M SyndromeUnited 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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Kyowa Kirin Co., Ltd.Active, not recruitingX-linked Hypophosphatemia (XLH)China
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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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Rare Thyroid Therapeutics International ABPremier Research Group plc; Egetis TherapeuticsRecruitingMonocarboxylate Transporter 8 Deficiency | Allan-Herndon-Dudley SyndromeUnited States, United Kingdom, Netherlands
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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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Children's Hospital Medical Center, CincinnatiActive, not recruitingMusculoskeletal Abnormalities | Duchenne Muscular DystrophyUnited States
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Kyowa Kirin Pharmaceutical Development LtdRecruitingX-Linked HypophosphatemiaFrance, Netherlands, United 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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Rare Thyroid Therapeutics International ABEgetis Therapeutics; AnovoRxAvailableMonocarboxylate Transporter 8 Deficiency | Allan-Herndon-Dudley SyndromeUnited States
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Mayo ClinicCompleted
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Stealth BioTherapeutics Inc.CompletedBarth SyndromeUnited States
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St. Jude Children's Research HospitalBaylor College of Medicine; Children's Hospital of Philadelphia; Dana-Farber... and other collaboratorsRecruitingMyelodysplastic Syndromes | Sickle Cell Disease | Fanconi Anemia | Severe Congenital Neutropenia | Bone Marrow Failure Syndromes | Blood Coagulation Disorder | Erythrocyte Disorder | Leukocyte Disorder | Hemostasis | Dyskeratosis Congenita | Diamond-Blackfan Anemia | Congenital Thrombocytopenia | Myeloproliferative...United States
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Ain Shams UniversityRecruiting
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Washington University School of MedicineCompleted
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Kyowa Kirin Co., Ltd.Active, not recruitingX-linked Hypophosphatemia (XLH)China
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Uppsala UniversityBritish Medical Research Council; Uppsala University HospitalCompletedAdrenocortical Carcinoma | Adrenal Cushing Syndrome | Primary Aldosteronism Due to Aldosterone Producing Adenoma | Primary Aldosteronism Due to Nodular Hyperplasia | Non-Secretory Adrenal AdenomaSweden
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Ionis Pharmaceuticals, Inc.RecruitingMethyl CpG Binding Protein 2 (MECP2) Duplication SyndromeUnited 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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Shenzhen Second People's HospitalGuangzhou Women and Children's Medical Center; Shenzhen UniversityRecruitingAdrenoleukodystrophy | Metachromatic LeukodystrophyChina
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BiogenCompletedChoroideremiaUnited States, Germany, France
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Duke UniversityFoundation Fighting BlindnessWithdrawnChoroideremiaUnited States
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University of AlbertaCanadian Institutes of Health Research (CIHR); Imperial College London; University... and other collaboratorsCompleted