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Clinical Trials on X-linked Spinocerebellar Ataxia Type 3
Total 124 results
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University of California, San DiegoRocket Pharmaceuticals Inc.Recruiting
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Rocket Pharmaceuticals Inc.Completed
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Lundquist Institute for Biomedical Innovation at...CompletedMucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type VIUnited States
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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Brazil
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JCR Pharmaceuticals Co., Ltd.Active, not recruitingMucopolysaccharidosis IIJapan
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JCR Pharmaceuticals Co., Ltd.Completed
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AO GENERIUMRecruitingMetabolic Diseases | Mucopolysaccharidosis Type IIRussian Federation
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Denali Therapeutics Inc.Enrolling by invitationMucopolysaccharidosis IIUnited States, Canada, Belgium, Czechia, Netherlands
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JCR Pharmaceuticals Co., Ltd.Enrolling by invitationMucopolysaccharidosis IIUnited States
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Rocket Pharmaceuticals Inc.Active, not recruitingDanon DiseaseUnited States
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REGENXBIO Inc.CompletedMucopolysaccharidosis IIBrazil, United States, United Kingdom
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedMucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type VI | Mucopolysaccharidosis Type IV | Mucopolysaccharidosis Type VIIUnited States, Canada
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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Canada
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University Hospital HeidelbergCompletedMucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Coping Behavior | Mucopolysaccharidosis Type III | Behavior DisordersGermany
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Rocket Pharmaceuticals Inc.RecruitingDanon DiseaseUnited States
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Denali Therapeutics Inc.RecruitingMucopolysaccharidosis IIUnited States, Spain, United Kingdom, Czechia, France, Canada, Italy, Belgium, Netherlands, Germany, Argentina, Sweden, Turkey, Brazil, Colombia
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AO GENERIUMCompletedA Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy VolunteersMetabolic Diseases | Mucopolysaccharidosis Type IIRussian Federation
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TakedaCompletedMucopolysaccharidosis (MPS)Brazil
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Fairview University Medical CenterTerminatedImmunologic Deficiency Syndromes | Common Variable Immunodeficiency | Severe Combined Immunodeficiency | Chronic Granulomatous Disease | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | Graft Versus Host Disease | X-linked Agammaglobulinemia | Chediak-Higashi Syndrome | Virus-Associated... and other conditionsUnited 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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Manchester University NHS Foundation TrustCompletedMucopolysaccharidosis Type IIUnited Kingdom
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H. Lee Moffitt Cancer Center and Research InstituteRecruitingLynch Syndrome | Pancreatic Ductal Adenocarcinoma | Chronic Pancreatitis | Pancreatic Cyst | Ataxia Telangiectasia | FAP | Peutz-Jeghers Syndrome | PALB2 Gene Mutation | BRCA Mutation | Fatty Pancreas | Genetic Pancreatic Cancer | Genetic Pancreatitis | Familial Atypical Multiple Mole-MelanomaUnited States
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Cairo UniversityCompletedMuscular Dystrophy, Duchenne TypeEgypt
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University of ArkansasWithdrawnPancreatic Neoplasms | Colorectal Neoplasms, Hereditary Nonpolyposis | BRCA1 Gene Mutation | BRCA2 Gene Mutation | Hereditary Pancreatitis | Ataxia Telangiectasia | Peutz-Jegher's Syndrome | Familial Atypical Mole-Malignant Melanoma SyndromeUnited States
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Bio Products LaboratoryCompletedCommon Variable Immunodeficiency | Wiskott-Aldrich Syndrome | X-linked Agammaglobulinemia | Primary Immune Deficiency Disorders | Hyper-IgM SyndromeUnited States, Chile, Israel
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West Kazakhstan Medical UniversityRecruitingOrnithine Transcarbamylase Deficiency | Biotinidase Deficiency | Citrullinemia | Glutaric Acidemia Type II | Argininosuccinic Aciduria | Maple Syrup Urine Disease | Primary Carnitine Deficiency | Homocystinuria | Carnitine Palmitoyltransferase II Deficiency | Arginase Deficiency | Very Long-chain Acyl-CoA... and other conditionsKazakhstan
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University of ChicagoNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedKrabbe Disease | Mucopolysaccharidosis Type II (MPS II) | Mucopolysaccharidosis Type I (MPS I) | Mucopolysaccharidosis Type III (MPS III) | Mucopolysaccharidosis Type VI (MPS VI)United States
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University of MalagaCompletedMuscular Dystrophy, Duchenne and Becker TypesSpain
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Politecnico di MilanoIRCCS Eugenio Medea; Villa Beretta Rehabilitation CenterCompletedMuscular Dystrophies | Muscular Dystrophy, Duchenne | Muscular Dystrophy, Becker | Muscular Dystrophy, Limb-Girdle Type 2Italy
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Rigshospitalet, DenmarkCompletedBecker Muscular Dystrophy | Limb-Girdle Muscular Dystrophy Type 2IDenmark
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Astellas Pharma Global Development, Inc.No longer availableAcute Myeloid Leukemia (AML) | FMS-like Tyrosine Kinase-3 (FLT3) MutationsAustralia, Italy, United Kingdom
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Astellas Pharma Global Development, Inc.RecruitingAcute Myeloid Leukemia (AML) | Acute Myeloid Leukemia With FMS-like Tyrosine Kinase 3 (FLT3) Mutation / Internal Tandem Duplication (ITD)United States, Canada, Germany, Italy, Spain, United Kingdom, France
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Baylor College of MedicineThe Methodist Hospital Research Institute; Center for Cell and Gene Therapy...CompletedAcute Myeloid Leukemia | Acute Lymphoblastic Leukemia | Myelodysplastic Syndrome | Chronic Myelogenous Leukemia | Hemophagocytic Lymphohistiocytosis (HLH) | Non Hodgkins Lymphoma | Familial Hemophagocytic Lymphohistiocytosis (FLH) | Viral-associated Hemophagocytic Syndrome (VAHS) | X-linked Lymphoproliferative...United 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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Mads Peter Godtfeldt StemmerikEdgewise Therapeutics, Inc.CompletedBecker Muscular Dystrophy | McArdle Disease | Limb-Girdle Muscular Dystrophy Type 2Denmark
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National Institute of Allergy and Infectious Diseases...TerminatedEctodermal Dysplasia | Hyper-IgM SyndromeUnited States
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University of California, San FranciscoRecruitingMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI | Wolman Disease | Pompe Disease Infantile-Onset | Mucopolysaccharidosis IV A | Mucopolysaccharidosis VII | Neuronopathic Gaucher DiseaseUnited States
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University of Milano BicoccaComitato Maria Letizia Verga,ItalyRecruitingLymphoma, Non-Hodgkin | Leukemia, Myeloid, Acute | Leukemia, B-cell | Adrenoleukodystrophy | Lymphoma, Hodgkin | Thalassemia Major | Leukemia, T Cell | DrepanocytosisItaly
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Astellas Pharma Global Development, Inc.Approved for marketingAcute Myeloid Leukemia (AML) | FMS-like Tyrosine Kinase-3 (FLT3) MutationsUnited States, Canada, Japan
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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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Mendel TuchmanChildren's Hospital of Philadelphia; University of California, Los Angeles; Icahn... and other collaboratorsCompletedMethylmalonic Acidemia | Carbamoyl-Phosphate Synthase I Deficiency Disease | Propionic Acidemia, Type I and/or Type II | Ornithine Carbamoyltransferase DeficiencyUnited 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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Turkish Society of Pediatric Gastroenterology,...CompletedDuchenne Muscular Dystrophy | Pompe Disease (Late-onset)Turkey
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Masonic Cancer Center, University of MinnesotaCompletedNiemann-Pick Disease | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | GM1 Gangliosidosis | Fucosidosis | Globoid Cell Leukodystrophy | Gaucher's Disease | Tay Sachs Disease | Sandhoff 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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Ultragenyx Pharmaceutical IncTerminatedWilson Disease | Ornithine Transcarbamylase Deficiency | Glycogen Storage Disease Type IASpain, United Kingdom, Canada, United States, Brazil
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Joanne Kurtzberg, MDThe Marcus FoundationRecruitingMucopolysaccharidosis II | Brain Diseases, Metabolic, Inborn | Adrenoleukodystrophy | Batten Disease | Leukodystrophy, Metachromatic | Leukodystrophy, Globoid Cell | Alpha-Mannosidosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | Neimann Pick Disease | Sanfilippo MucopolysaccharidosesUnited States
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Assistance Publique - Hôpitaux de ParisNot yet recruitingHemophilia A | Hemophilia B | Cystic Fibrosis | Sickle Cell Disease | Muscular Dystrophy, Duchenne | Fragile X Syndrome | Huntington Disease | Myotonic Dystrophy | Autosomal Recessive Polycystic Kidney Disease | Neurofibromatosis-Noonan Syndrome | Muscular Dystrophy, Becker | Invasive PreNatal Diagnosis in a... and other conditionsFrance
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M.A.G.I.C. Clinic LTDHanalytics Solutions Inc.RecruitingMitochondrial Diseases | Fabry Disease | Metabolic Disease | Gaucher Disease | Pompe DiseaseCanada
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Albert Einstein College of MedicineEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationMucopolysaccharidosis II | Fabry Disease | Niemann-Pick Disease, Type C | Mucopolysaccharidosis VI | Lysosomal Acid Lipase Deficiency | Metachromatic Leukodystrophy | Gaucher Disease | Cerebrotendinous Xanthomatosis | Mucopolysaccharidosis IV A | GM1 Gangliosidosis | Mucopolysaccharidosis VII | Acid Sphingomyelinase... and other conditionsUnited States