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Clinical Trials on Bulbo-Spinal Atrophy, X-Linked
Total 1856 results
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Yonsei UniversityRecruitingFabry DiseaseKorea, Republic of
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Yonsei UniversityRecruitingFabry DiseaseKorea, Republic of
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Yonsei UniversityRecruitingFabry's DiseaseKorea, Republic of
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Genzyme, a Sanofi CompanyTerminatedFabry Disease | Alpha Galactosidase A DeficiencyUnited States, Austria, United Kingdom
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Resverlogix CorpWithdrawn
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Kyowa Kirin Co., Ltd.CompletedA Study of KRN23 in Adult and Pediatric Patients With X-linked Hypophosphatemic Rickets/OsteomalaciaXLHJapan, Korea, Republic of
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University Children's Hospital, ZurichCompletedPhenylketonurias | OTC Deficiency | Inborn Errors of Metabolism | Urea Cycle Disorder | Maple Syrup Urine Disease | Methylmalonicacidemia | Aminoacidopathy | Patient Reported Outcome MeasurementsSwitzerland
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University of Maryland, BaltimoreCompletedOsteoporosis PseudogliomaUnited States
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Ulla Feldt-RasmussenCompleted
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Wolfson Medical CenterUnknownFabry Disease in the Young StrokeIsrael
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Genzyme, a Sanofi CompanyWithdrawn
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National Institute of Neurological Disorders and...CompletedHealthy | Fabry Disease | Cerebrovascular AccidentUnited States
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University of FloridaLive Like Bella Pediatric Cancer ResearchCompletedCommon Variable Immunodeficiency | Severe Combined Immunodeficiency | Sickle Cell Disease | Hurler Syndrome | Chronic Granulomatous Disease | Thalassemia | Wiskott-Aldrich Syndrome | Adrenoleukodystrophy | X-linked Lymphoproliferative Disease | Diamond Blackfan Anemia | Bone Marrow Failure Syndrome | Hemophagocytic... and other conditionsUnited States
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Anagnostou, Evdokia, M.D.The Hospital for Sick Children; Canadian Institutes of Health Research (CIHR); Alberta Health services and other collaboratorsNot yet recruitingAnxiety Disorders | Generalized Anxiety Disorder | Anxiety | Tuberous Sclerosis | ADHD | Neurodevelopmental Disorders | Autism Spectrum Disorder | Fragile X Syndrome | Social Anxiety | Social Anxiety Disorder | Agoraphobia | Tourette Syndrome | Tic Disorders | Autism | ADHD Predominantly Inattentive Type | ADHD - Combined... and other conditionsCanada
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University of WashingtonCompletedMultiple Sclerosis | Muscular Dystrophy | Spinal Cord Injury | Post-polio SyndromeUnited States
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Kahramanmaras Sutcu Imam UniversityCompletedCerebral Palsy | Muscular Dystrophy | Caregiver Burden | Down Syndrome | Spina Bifida | Disabilities MultipleTurkey
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iECURE, Inc.RecruitingUrea Cycle Disorders, Inborn | Ornithine Transcarbamylase Deficiency | Ornithine Transcarbamylase Deficiency Disease | Ornithine Carbamoyltransferase Deficiency (Disorder)United Kingdom
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Yonsei UniversityRecruitingFabry DiseaseKorea, Republic of
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Netherlands, Hungary, United Kingdom, Czechia, Norway, Slovenia, Spain, Finland, France, Italy, Switzerland
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NYU Langone HealthNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)CompletedCystinuria | Primary Hyperoxaluria | Dent Disease | Adenine Phosphoribosyl Transferase DeficiencyUnited States, Iceland
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Novo Nordisk A/SCompletedHealthy | Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors | Acquired Bleeding Disorder | Acquired Haemophilia | Congenital FVII Deficiency | Glanzmann's DiseaseFrance
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Lysosomal and Rare Disorders Research and Treatment...Recruiting
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Chiesi Farmaceutici S.p.A.Active, not recruitingFabry DiseaseUnited States, Netherlands, Australia, Hungary, United Kingdom, Canada, Czechia, Finland, France, Italy, Norway, Slovenia, Spain
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University of Maryland, BaltimoreChildren's Hospital of PhiladelphiaWithdrawnOsteoporosis Pseudoglioma SyndromeUnited States
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CENTOGENE GmbH RostockWithdrawnStroke, Acute | Cerebrovascular Accident | Cerebral StrokeGermany
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Rennes University HospitalCompleted
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University College, LondonUniversity of Sydney; University Hospital BirminghamUnknownFabry DiseaseUnited Kingdom, Australia
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Genzyme, a Sanofi CompanyCompletedFabry DiseaseUnited States, United Kingdom, Canada, Hungary, Poland, Czech Republic
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National Institute of Neurological Disorders and...CompletedFabry DiseaseUnited States
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Royal College of Surgeons, IrelandBeaumont Hospital; Post Polio Support Group IrelandCompleted
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Bastyr UniversityRecruitingParkinson's Disease | Parkinsonism | Corticobasal Degeneration | Shy-Drager Syndrome | Progressive Supranuclear Palsy | Dementia With Lewy Bodies | MSA - Multiple System Atrophy | Olivopontocerebellar Atrophies | Pick DiseaseUnited States
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University of CambridgeSanofiRecruiting
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Fundacion para la Investigacion Biomedica del Hospital...Fundación Mutua MadrileñaRecruiting
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Baylor College of MedicineChildren's Hospital of Philadelphia; Children's Hospital Colorado; Seattle Children... and other collaboratorsRecruitingOrnithine Transcarbamylase Deficiency | Urea Cycle Disorder | Argininosuccinic Aciduria | Hyperargininemia | Citrullinemia 1 | ARGI Deficiency | ASL Deficiency | ASS DeficiencyUnited States
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University Hospital, AngersRecruiting
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Chiesi Farmaceutici S.p.A.Active, not recruitingFabry DiseaseBelgium, United States, United Kingdom, Italy, Czechia, Denmark, Norway
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Assistance Publique - Hôpitaux de ParisShire International GmbHCompleted
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Children's Mercy Hospital Kansas CityCompletedHypophosphatemic Rickets, X-Linked DominantUnited States
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National Human Genome Research Institute (NHGRI)Completed
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University Hospital, BordeauxCompletedPain | Fabry's DiseaseFrance
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National Institute of Neurological Disorders and...CompletedHealthy | Postpoliomyelitis Syndrome | Neuromuscular DiseaseUnited States
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Masonic Cancer Center, University of MinnesotaRecruitingCommon Variable Immunodeficiency | Chronic Granulomatous Disease | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | X-linked Lymphoproliferative Disease | Langerhan's Cell Histiocytosis | Chediak-Higashi Syndrome | Griscelli Syndrome | Bare Lymphocyte Syndrome | SCID | Omenn's Syndrome | Reticular... and other conditionsUnited States
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Wuerzburg University HospitalCharite University, Berlin, Germany; University Hospital Muenster; Health Care...RecruitingQuality of Life | Adherence, Medication | Fabry Disease | Rare DiseasesGermany
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Mayo ClinicNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingCystinuria | Primary Hyperoxaluria | Dent Disease | APRT DeficiencyUnited States, Iceland
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Ultragenyx Pharmaceutical IncKyowa Kirin Co., Ltd.AvailableX-linked Hypophosphatemia | Tumor-Induced Osteomalacia
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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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Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-linked HypophosphatemiaNetherlands, United States, United Kingdom, France
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Orthopedic Hospital Vienna SpeisingCompleted
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University of MinnesotaGenzyme, a Sanofi CompanyWithdrawnNeuropathic Pain | Fabry DiseaseUnited States