Search clinical trials for: Niemann-Pick disease type C
Total 495009 results
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Chung-Hsing WangSanofiNot yet recruiting
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SanofiPulse Infoframe Ltd.RecruitingNiemann-Pick Diseases | Acid Sphingomyelinase DeficiencyUnited States
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Fundación Española de Hematología y HemoterapíaRecruitingGaucher Disease | Acid SphingoMyelinase DeficiencySpain
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Eline C. B. EskesRecruiting
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Eline C. B. EskesRecruitingAsmd, Visceral TypeNetherlands
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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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Fundación Española de Hematología y HemoterapíaRecruitingNiemann-Pick Diseases | Gaucher Disease | Splenomegaly | Acid SphingoMyelinase Deficiency | ASMDSpain
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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease, Type CUnited States
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SanofiRecruitingAcid Sphingomyelinase Deficiency (ASMD)France
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Mansoura UniversityNot yet recruitingOveractive Bladder | Voiding Disorders
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Fondazione I.R.C.C.S. Istituto Neurologico Carlo...RecruitingNeurologic Disorder | Rare Diseases | Metabolic Disease | Inherited Disease | Undiagnosed Disease | Neuro-Degenerative DiseaseItaly
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Cyclo Therapeutics, Inc.RecruitingNiemann-Pick Disease, Type C1United States, Spain, Israel, Taiwan, Brazil, Germany, Australia, Poland, Turkey, Italy, United Kingdom
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SanofiRecruitingAcid SphingoMyelinase Deficiency | Gaucher Disease, SplenomegalyFrance
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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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University of PittsburghRecruitingLysosomal Storage Diseases | Leukodystrophy | Niemann-Pick Diseases | Batten Disease | Gaucher Disease | Krabbe Disease | Alpha-Mannosidosis | Osteopetrosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | GM1 Gangliosidoses | MPS I | MPS II | Vanishing White Matter Disease | Multiple Sulfatase Deficiency... and other conditionsUnited States
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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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Masonic Cancer Center, University of MinnesotaRecruitingHurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Metachromatic Leukodystrophy | Alpha-Mannosidosis | Hunter Syndrome | Mucopolysaccharidosis Disorders | Maroteaux Lamy Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Glycoprotein Metabolic Disorders | Recessive Leukodystrophies | Globoid... and other conditionsUnited States
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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease, Type CUnited States
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ZevraDenmarkActive, not recruitingNiemann-Pick Disease, Type CUnited States, Spain, United Kingdom, Denmark, France, Germany, Italy, Poland, Switzerland
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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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Azafaros A.G.Active, not recruitingNiemann-Pick Disease, Type C | GM2 GangliosidosisBrazil
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Mandos LLCTerminatedNiemann-Pick Type C DiseaseUnited States
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Mandos LLCTerminatedNiemann-Pick Disease, Type CCosta Rica
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Genzyme, a Sanofi CompanyCompletedSphingomyelin LipidosisUnited States, Argentina, Australia, Belgium, Brazil, Bulgaria, Chile, France, Germany, Italy, Japan, Netherlands, Portugal, Spain, Tunisia, Turkey, United Kingdom
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IntraBio IncCompletedNiemann-Pick Disease, Type CUnited States, United Kingdom, Germany, Slovakia, Spain
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ZevraDenmarkAvailableNiemann-Pick Disease, Type CUnited States
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Genzyme, a Sanofi CompanyCompletedSphingomyelin LipidosisUnited States, Belgium, Brazil, France, Germany, Italy, United Kingdom
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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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Mandos LLCTerminatedNiemann-Pick Disease, Type CUnited States, Australia, United Kingdom, Turkey, Switzerland, Germany, New Zealand, Singapore, France
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SanofiCompletedSphingomyelin LipidosisItaly, Czechia, Belgium, France, Portugal, Turkey, United States, Argentina, Brazil, Chile, Germany, Romania, Spain
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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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Talaris Therapeutics Inc.Duke UniversityTerminatedNiemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...United States
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Mandos LLCCompletedNiemann-Pick Disease, Type CUnited States, Australia, United Kingdom, Germany, France, New Zealand, Singapore, Spain, Turkey
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CENTOGENE GmbH RostockWithdrawnNiemann-Pick Disease, Type C | Niemann-Pick DiseaseGermany, India, Sri Lanka
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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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SanofiApproved for marketing
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Eunice Kennedy Shriver National Institute of Child...TerminatedNiemann-Pick Disease, Type C1United States
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ActelionCompleted
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Genzyme, a Sanofi CompanyCompletedSphingomyelin LipidosisBrazil, France, Italy, United States, Germany, United Kingdom
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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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Melbourne HealthSanofiWithdrawnLysosomal Storage Diseases | Fabry Disease | Niemann-Pick Disease, Type C | Gaucher DiseaseAustralia
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CENTOGENE GmbH RostockCompleted
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Cyclo Therapeutics, Inc.CompletedNiemann-Pick Disease, Type C1United States
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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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Vtesse, LLC, a Mallinckrodt Pharmaceuticals CompanyWithdrawnNiemann-Pick Disease, Type C
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Alexion PharmaceuticalsTerminatedLysosomal Acid Lipase DeficiencyUnited Kingdom, Finland, United States, Italy
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Xinhua Hospital, Shanghai Jiao Tong University...UnknownNiemann-Pick Disease, Type C1China
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Alexion PharmaceuticalsCompletedWolman Disease | Lysosomal Acid Lipase DeficiencyUnited Kingdom, France, United States, Egypt, Ireland