Clinical Trials on Mannosidosis, Beta A, Lysosomal
Total 138 results
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Melbourne HealthSanofiWithdrawnLysosomal Storage Diseases | Fabry Disease | Niemann-Pick Disease, Type C | Gaucher DiseaseAustralia
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Cyclo Therapeutics, Inc.CompletedNiemann-Pick Disease, Type C1United States
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University Hospital, GrenobleTerminatedPsychiatric Adults PatientsFrance
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Mayo ClinicNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedNiemann-Pick Disease, Type CUnited States
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Eunice Kennedy Shriver National Institute of Child...Washington University School of Medicine; University of Oxford; National Human...CompletedNiemann-Pick Disease, Type CUnited States
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National Institute of Neurological Disorders and...CompletedBrain Neoplasm | Alzheimer's Disease | Niemann Pick DiseaseUnited States
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IntraBio IncCompletedNiemann-Pick Disease, Type CUnited States, United Kingdom, Germany, Slovakia, Spain
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Eunice Kennedy Shriver National Institute of Child...TerminatedNiemann-Pick Disease, Type C1United States
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Cyclo Therapeutics, Inc.CompletedNiemann-Pick Disease, Type C1Israel, Sweden, United Kingdom
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Vtesse, LLC, a Mallinckrodt Pharmaceuticals CompanyCompletedNiemann-Pick Disease, Type C1United States
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Cyclo Therapeutics, Inc.Active, not recruitingNiemann-Pick Disease, Type C1United States
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CENTOGENE GmbH RostockCompleted
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KemPharm Denmark A/SCompletedNiemann-Pick Disease, Type CSpain, France, United Kingdom, Switzerland, Denmark, Poland, Italy, Germany
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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease, Type CUnited States
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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease, Type CUnited States
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IntraBio IncActive, not recruitingNiemann-Pick Disease, Type CUnited States, Australia, United Kingdom, Germany, Slovakia, Netherlands, Czechia, Switzerland
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National Niemann-Pick Disease FoundationIcahn School of Medicine at Mount Sinai; Engage Health Inc.CompletedNiemann-Pick DiseasesUnited States
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Eunice Kennedy Shriver National Institute of Child...Washington University School of Medicine; Weill Medical College of Cornell...CompletedNeimann-Pick DiseaseUnited States
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Genzyme, a Sanofi CompanyTerminatedNiemann-Pick Disease | Acid Sphingomyelinase DeficiencyUnited States
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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
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National Taiwan University HospitalCompleted
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Cyclo Therapeutics, Inc.Active, not recruitingNiemann-Pick Disease, Type C1United States, Taiwan, United Kingdom, Israel, Spain, Brazil, Italy, Australia, Turkey, Saudi Arabia, Argentina, Poland, Germany
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Washington University School of MedicineEunice Kennedy Shriver National Institute of Child Health and Human Development...RecruitingNiemann-Pick Disease, Type CUnited States
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Fundación Española de Hematología y HemoterapíaRecruitingGaucher Disease | Acid SphingoMyelinase DeficiencySpain
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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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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease Type C1 | Juvenile Neuronal Ceroid Lipofuscinosis | Smith-Lemli-Opitz Syndrome | Creatine Transporter DeficiencyUnited States
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Sangamo TherapeuticsActive, not recruitingHemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis IIUnited States
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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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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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Children's Hospital Los AngelesCompletedGranuloma | Anemia | Thrombocytopenia | Neutropenia | Sickle Cell Disease | Thalassemia | Niemann-Pick Disease | Wiskott-Aldrich Syndrome | Osteopetrosis | Fucosidosis | Chediak Higashi Syndrome | Hurler DiseaseUnited 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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Sanguine BiosciencesRecruitingHeart Diseases | Stroke | Lymphoma | Multiple Sclerosis | Kidney Diseases | Diabetes | Leukemia | Age-Related Macular Degeneration | Cancer | Arthritis | Schizophrenia | Thyroid Diseases | Parkinson Disease | Hepatitis | Vasculitis | Psoriasis | Asthma | Amyloidosis | Fibromyalgia | Graves Disease | Alzheimer Disease | Celiac Disease | Cirr... and other conditionsUnited States
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Emory UniversityCompletedSickle Cell Disease | Fanconi Anemia | Hurler Syndrome | Chronic Granulomatous Disease | Severe Congenital Neutropenia | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Glanzmann Thrombasthenia | Shwachman-Diamond Syndrome | Thalassemia Major | Leukocyte... and other conditionsUnited States
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New York Medical CollegeCompletedMucopolysaccharidosis I | Myelodysplastic Syndrome | Acute Lymphocytic Leukemia | Acute Myelogenous Leukemia | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Fucosidosis | Gaucher... and other conditionsUnited States
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Sanford HealthNational Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaboratorsRecruitingMitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditionsUnited States, Australia
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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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Centre Hospitalier Universitaire de LiegeSanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaboratorsRecruitingCongenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditionsBelgium
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UK Kidney AssociationRecruitingVasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditionsUnited Kingdom