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Search clinical trials for: glycosphingolipid storage disorder
Total 148661 results
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National Institute of Neurological Disorders and...Recruiting
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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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AlexionRecruitingWolman Disease | Cholesterol Ester Storage Disease | Lysosomal Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 | Acid Lipase Deficiency | LIPA Deficiency | LAL-DeficiencyFrance, Belgium, United States, Spain, Germany, Greece, Israel, Italy, Slovenia, United Kingdom, Brazil, Canada, Denmark, Australia, Croatia, Czechia, Ireland, Mexico, Netherlands, Poland, Portugal, Saudi Arabia
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Washington... and other collaboratorsRecruiting
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St. Jude Children's Research HospitalRecruitingMucopolysaccharidosis I | Severe Combined Immunodeficiency | Neuroblastoma | Neoplasm | Malignancy, HematologicUnited States
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Late Onset Tay-Sachs DiseaseUnited States
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Canadian Fabry Research ConsortiumNova Scotia Health AuthorityRecruiting
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Genzyme, a Sanofi CompanyRecruitingGaucher Disease | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency DiseaseHungary, Croatia, United States, Australia, Belgium, Brazil, Bulgaria, Canada, Denmark, Germany, Greece, Hong Kong, India, Indonesia, Italy, Jordan, Korea, Republic of, Kuwait, Lebanon, Malaysia, Pakistan, Philippines, Poland, Saudi Arabia and more
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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease, Type CUnited States
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Genzyme, a Sanofi CompanyRecruitingFabry DiseaseCroatia, United States, Argentina, Australia, Belgium, Bulgaria, Canada, Germany, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Malaysia, Philippines, Poland, Romania, Singapore, Slovakia, Taiwan, Thailand, United Kingdom, Vietn... and more
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National Human Genome Research Institute (NHGRI)RecruitingPulmonary Fibrosis | Healthy Volunteers | Hermansky-Pudlak Syndrome (HPS)United States
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Eunice Kennedy Shriver National Institute of Child...RecruitingLysosomal Storage Disease | Cholesterol MetabolismUnited States
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National Human Genome Research Institute (NHGRI)Recruiting
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National Human Genome Research Institute (NHGRI)RecruitingHermansky-Pudlak Syndrome (HPS)United States
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ShireTerminatedFabry DiseaseSpain, Greece, Germany, Portugal, Canada, Finland, Poland, Sweden
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National Human Genome Research Institute (NHGRI)Enrolling by invitationParkinson Disease | Gaucher Disease | Lysosomal Storage DisordersUnited States
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Hoffmann-La RocheGOG Foundation; European Network of Gynaecological Oncological Trial Groups...Active, not recruitingOvarian CancerUnited States, Korea, Republic of, France, Germany, Spain, Italy, Canada, Russian Federation, United Kingdom, Turkey, Australia, Czechia, Switzerland
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Wuerzburg University HospitalTakedaActive, not recruitingLysosomal Storage Diseases | Fabry Disease | Fabry Disease, Cardiac Variant | HCM - Hypertrophic Cardiomyopathy | Anderson Fabry DiseaseGermany
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University of California, San DiegoCalifornia Institute for Regenerative Medicine (CIRM); Cystinosis Research...Active, not recruiting
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Columbia UniversityNational Heart, Lung, and Blood Institute (NHLBI); New York Blood CenterCompletedIron DeficiencyUnited States
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Denali Therapeutics Inc.CompletedMucopolysaccharidosis IIUnited Kingdom, United States, Netherlands
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National Human Genome Research Institute (NHGRI)CompletedParkinson Disease | Gaucher DiseaseUnited States
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ShireTakeda Development Center Americas, Inc.Active, not recruitingMetachromatic Leukodystrophy (MLD)United States, Canada, Belgium, Israel, Netherlands, United Kingdom, Germany, Spain, Japan, Brazil, Argentina, France, Greece, Italy
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Forge Biologics, IncActive, not recruiting
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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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University of PittsburghWithdrawnMetachromatic LeukodystrophyUnited States
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Ultragenyx Pharmaceutical IncCompletedGlycogen Storage Disease Type IANetherlands, United States
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Passage Bio, Inc.Active, not recruitingGM1 Gangliosidosis | GM1 Gangliosidosis, Type I | GM1 Gangliosidosis, Type 2 | Beta-Galactosidase-1 (GLB1) DeficiencyUnited States, Canada, United Kingdom, Turkey, Brazil
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Denali Therapeutics Inc.Enrolling by invitationMucopolysaccharidosis IIUnited States, Canada, Belgium, Czechia, Netherlands
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Amicus Therapeutics France SASCompletedFabry Disease | Anderson Fabry DiseaseFrance
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Children's Hospital of PhiladelphiaIllumina, Inc.Active, not recruitingMucopolysaccharidoses | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2 Gangliosidosis | Zellweger... and other conditionsUnited States
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Amicus Therapeutics France SASCompleted
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Duke UniversitySuspendedLysosomal Storage Diseases | Genetic Disease | Inborn Errors of Metabolism | Glycogen Storage Disease | Storage DiseaseUnited 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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University Hospital Birmingham NHS Foundation TrustNorthern Care Alliance NHS Foundation Trust; Cambridge University Hospitals... and other collaboratorsActive, not recruitingArrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease (RaILRoAD)Fabry DiseaseUnited Kingdom, Australia
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CinnagenCompletedMucopolysaccharidosis Type 1Iran, Islamic Republic of
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University of AarhusThe Danish Dairy Research Foundation, DenmarkCompleted
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Sangamo TherapeuticsActive, not recruitingFabry DiseaseUnited States, United Kingdom, Taiwan, Australia, Germany, Italy, Canada
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ShireActive, not recruiting
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X4 PharmaceuticalsActive, not recruiting
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ShireCompletedHunter SyndromeUnited States, United Kingdom, Canada
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ShireCompletedHunter SyndromeUnited States, Spain, Canada, United Kingdom, Mexico, Australia, France
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PfizerArena is a wholly owned subsidiary of PfizerCompletedEosinophilic EsophagitisUnited States, Spain, Switzerland, Australia, Belgium, Netherlands, Canada, Germany
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University of PittsburghNational Institute of Neurological Disorders and Stroke (NINDS)Active, not recruiting
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Ultragenyx Pharmaceutical IncActive, not recruitingGlycogen Storage Disease Type IA | Von Gierke's Disease (GSD Type Ia)Netherlands, United States, Canada, Spain
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Sangamo TherapeuticsActive, not recruitingHemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis IIUnited States
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Vastra Gotaland RegionKarolinska University Hospital; Göteborg University; Uppsala University HospitalCompleted
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Denali Therapeutics Inc.Active, not recruitingMucopolysaccharidosis IIUnited States, Netherlands, Canada, United Kingdom
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Massachusetts General HospitalActive, not recruiting