Clinical Trials on Lysosomal Storage Disorders
Total 150409 results
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Tehran University of Medical SciencesMashhad University of Medical Sciences; Kashan University of Medical SciencesRecruitingEffects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) (EMTISTD)Supportive Care | GM2 GangliosidosisIran, Islamic Republic of
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Cambridge University Hospitals NHS Foundation TrustMedical Research Council; National Institute for Health Research, United KingdomRecruitingGaucher Disease, Type III | Gaucher Disease, Type IUnited Kingdom
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SanofiCompleted
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SanofiCompletedGaucher Disease | SplenomegalyFrance
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Shaare Zedek Medical CenterCompleted
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Lysosomal and Rare Disorders Research and Treatment...UnknownGaucher Disease Type 1United States
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Amicus TherapeuticsCompletedGaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher DiseaseUnited States
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Amicus TherapeuticsCompletedGaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher DiseaseUnited Kingdom, Israel, South Africa, United States
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The Hospital for Sick ChildrenActelionCompletedGangliosidoses GM2Canada
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Rambam Health Care CampusUnknown
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National Center for Research Resources (NCRR)University of PittsburghUnknownGaucher's DiseaseUnited States
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Fondazione Don Carlo Gnocchi OnlusAssociazione Italiana Glicogenosi (AIG); Associazione Riabilitatori Insufficienza... and other collaboratorsRecruitingGlycogen Storage Disease Type II | Pompe Disease (Late-onset)Italy
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National Institute of Neurological Disorders and...Completed
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Massachusetts General HospitalActive, not recruiting
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SanofiCompleted
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AVROBIOWithdrawn
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NYU Langone HealthGenzyme, a Sanofi CompanyCompleted
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Amicus TherapeuticsCompletedGaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher DiseaseUnited Kingdom, United States
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SanofiCompletedGaucher DiseaseUnited States
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The Hospital for Sick ChildrenCompletedGangliosidoses, GM2 | Tay-Sachs Disease | Sandhoff DiseaseCanada
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National Institute of Neurological Disorders and...CompletedGaucher's DiseaseUnited States
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National Center for Research Resources (NCRR)National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); University...Completed
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Lysosomal and Rare Disorders Research and Treatment...RecruitingBone Diseases | Gaucher DiseaseUnited States
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Freeline TherapeuticsRecruitingGaucher Disease, Type 1Spain, Israel, United States, United Kingdom, Brazil, Germany, Paraguay
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IntraBio IncCompletedGM2 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Germany, Spain, United Kingdom
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Terence FlotteMassachusetts General Hospital; University of Massachusetts, WorcesterActive, not recruitingTay-Sachs Disease | Sandhoff DiseaseUnited States
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National Institute of Neurological Disorders and...CompletedGaucher DiseaseUnited States, United Kingdom
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Turkish Society of Pediatric Gastroenterology,...CompletedDuchenne Muscular Dystrophy | Pompe Disease (Late-onset)Turkey
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National Cerebral and Cardiovascular CenterMinistry of Health, Labor and Welfare (Japan)TerminatedMyocardial Infarction | Impaired Glucose ToleranceJapan
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Institut National de la Santé Et de la Recherche...Recruiting
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University Hospital Center of MartiniqueRecruiting
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University of California, IrvineCompletedMuscle Weakness | Pompe DiseaseUnited States
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Children's Hospital Medical Center, CincinnatiNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedWolman Disease | Cholesterol Ester Storage Disease | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2United States
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Centre Hospitalier Universitaire de NiceCompleted
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State University of New York at BuffaloEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsRecruiting
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGaucher Disease Type 1United States
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Genzyme, a Sanofi CompanyActive, not recruitingGaucher Disease Type 1 | Gaucher Disease Type 3Germany, United States, Japan, United Kingdom
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KemPharm Denmark A/STerminatedGaucher Disease, Type 1 | Gaucher Disease, Type 3India
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Baylor Research InstituteTexas Scottish Rite Hospital for ChildrenWithdrawnGaucher Disease Type 1 | Gaucher Disease Type 3United States
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The Cooper Health SystemCompletedMetachromatic LeukodystrophyUnited States
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Children's National Research InstituteActelionCompletedSandhoff Disease | GM2 Gangliosidoses | Tay-SachsUnited States
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National Human Genome Research Institute (NHGRI)CompletedParkinson Disease | Gaucher DiseaseUnited States
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Alexion PharmaceuticalsCompletedWolman Disease | Lysosomal Acid Lipase DeficiencyUnited Kingdom, France, United States, Egypt, Ireland
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Lysosomal and Rare Disorders Research and Treatment...UnknownParkinson Disease | Gaucher DiseaseUnited States
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Forge Biologics, IncNot yet recruitingKrabbe DiseaseUnited States
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University of PittsburghNational Institute of Neurological Disorders and Stroke (NINDS)Active, not recruiting
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Forge Biologics, IncActive, not recruiting
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Genzyme, a Sanofi CompanyActive, not recruitingTay-Sachs Disease | Sandhoff DiseaseUnited States, Argentina, Czechia, Portugal, Russian Federation, Turkey, United Kingdom, Brazil, France, Germany, Italy, Japan, Spain
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Orchard TherapeuticsOspedale San RaffaeleActive, not recruitingLysosomal Storage Diseases | Metachromatic LeukodystrophyItaly
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Azafaros A.G.Active, not recruitingGM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Brazil, France, Germany, Italy, United Kingdom