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Clinical Trials on Bulbo-Spinal Atrophy, X-Linked
Total 1856 results
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Kyowa Hakko Kirin Pharma, Inc.Kyowa Kirin Co., Ltd.CompletedX-linked HypophosphatemiaUnited States, Canada
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Amicus TherapeuticsApproved for marketingPhysician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry DiseaseFabry DiseaseUnited States, Australia
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Amicus TherapeuticsCompletedFabry DiseaseAustralia, Brazil
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Amicus TherapeuticsCompletedFabry DiseaseUnited States
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Amicus TherapeuticsTerminatedFabry DiseaseUnited Kingdom, United States, Australia, Brazil, France
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Amicus TherapeuticsCompletedFabry DiseaseFrance, United Kingdom
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Chiayi Christian HospitalSanofiUnknown
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National Institute of Neurological Disorders and...CompletedFabry DiseaseUnited States
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National Institute of Neurological Disorders and...Completed
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Spital LinthUniversity of Zurich; National University, SingaporeUnknown
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University Hospital, BordeauxUnknown
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O & O Alpan LLCCompletedFabry DiseaseUnited States
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors | Haemophilia A | Haemophilia B | Relatives to/Carers of PatientsSpain, Canada, United Kingdom
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors | Haemophilia A | Haemophilia BUnited States
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Ohio State UniversityGenzyme, a Sanofi CompanyCompleted
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Green Cross CorporationCompleted
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Cytonet GmbH & Co. KGCompletedOrnithine Transcarbamylase Deficiency | Urea Cycle Disorders | Citrullinemia | Carbamoylphosphate Synthetase I DeficiencyGermany
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Genzyme, a Sanofi CompanyCompletedFabry DiseaseUnited States, United Kingdom, Canada, Czech Republic, Hungary, Poland
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Mario Negri Institute for Pharmacological ResearchTerminated
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Amicus TherapeuticsCompleted
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National Institute of Neurological Disorders and...Completed
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National Institute of Neurological Disorders and...Completed
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University of PittsburghCompletedMuscular Dystrophy | ALS | Spinal Cord Injury | Tetraplegia | Brachial Plexus Injury | Brainstem StrokeUnited States
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Masonic Cancer Center, University of MinnesotaCompletedWolman Disease | Adrenoleukodystrophy | Metachromatic Leukodystrophy | Sanfilippo Syndrome | GM1 Gangliosidosis | Globoid Cell Leukodystrophy | Tay Sachs Disease | Sandhoffs Disease | I-Cell DiseaseUnited States
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University Health Network, TorontoUniversity of Colorado, Denver; Mayo Clinic; Icahn School of Medicine at Mount... and other collaboratorsRecruiting
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The University of QueenslandRecruitingPulmonary Fibrosis | Dyskeratosis Congenita | Telomere Shortening | Telomere DiseaseAustralia
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Canadian Fabry Research ConsortiumNova Scotia Health AuthorityRecruiting
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Sangamo TherapeuticsEnrolling by invitationFabry Disease | Fabry Disease, Cardiac VariantUnited States, Australia, United Kingdom
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Caroline Michaela KistorpRigshospitalet, Denmark; Amicus TherapeuticsActive, not recruiting
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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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University Hospital ErlangenCompletedHypohidrotic Ectodermal DysplasiaGermany
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Ospedale San DonatoAmicus Therapeutics; Institute of Biomedicine and Molecular Immunology - CNRCompletedHeart Diseases | Fabry DiseaseItaly
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University of WashingtonEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompletedMultiple Sclerosis | Low Back Pain | Chronic Pain | Muscular Dystrophies | Spinal Cord Injury | Other and Unspecified Amputation Stump ComplicationsUnited States
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First Affiliated Hospital of Fujian Medical UniversityRecruitingSpinocerebellar Ataxia Type 3 | Multiple System Atrophy-cerebellar TypeChina
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Sanko UniversityCompletedCerebral Palsy | Muscular Dystrophy | Down Syndrome | Parents | Spina BifidaTurkey
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CENTOGENE GmbH RostockWithdrawnFructose Metabolism, Inborn Errors | Glycogen Storage Disease Type II | Glycogen Storage Disease | Glycogen Storage Disease Type V | Glycogen Storage Disease Type I | Glycogen Storage Disease Type III | Glycogen Storage Disease Type VII | Glycogen Storage Disease Type IV | Glycogen Storage Disease Type... and other conditionsGermany, India, Sri Lanka
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Academisch Medisch Centrum - Universiteit van Amsterdam...RecruitingFabry Disease | Fabry Disease, Cardiac VariantNetherlands
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SanofiRecruitingFabry DiseaseItaly, United States, Austria, China, Czechia, Denmark, France, Germany, Greece, Japan, Korea, Republic of, Netherlands, Norway, Spain, Taiwan, Turkey, United Kingdom, Canada, Poland
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CENTOGENE GmbH RostockCompletedFabry Disease | Anderson-Fabry Disease | Fabry´s DiseaseArgentina, Belgium, Croatia, Czechia, Denmark, France, Germany, United Kingdom
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General University Hospital, PragueCharles University, Czech Republic; Amicus Therapeutics; Klinické centrum ISCAREUnknown
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National Human Genome Research Institute (NHGRI)TerminatedCitrullinemia | Argininosuccinic Aciduria | Hyperargininemia | Ornithine Carbamoyltransferase Deficiency | Carbamoyl-Phosphate Synthase I Deficiency | N-Acetylglutamate Synthase DeficiencyUnited States
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Ulla Feldt-RasmussenCompleted
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ProtalixWithdrawn
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Taipei Veterans General Hospital, TaiwanSanofiUnknownFabry Disease, Cardiac Variant
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University Hospital, CaenUnknownCardiomyopathiesFrance
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Cedars-Sinai Medical CenterNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsUnknown
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Masonic Cancer Center, University of MinnesotaCompletedMucopolysaccharidosis | Hurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Krabbe Disease | Adrenoleukodystrophy (ALD) | Hunter Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Metachromatic Leukodystrophy (MLD) | Maroteaux-Lamy SyndromeUnited 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