Clinical Trials on Inherited Metabolic Storage Diseases
Total 61455 results
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Shaare Zedek Medical CenterCompleted
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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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Freeline TherapeuticsRecruitingGaucher Disease, Type 1Spain, Israel, United States, United Kingdom, Brazil, Germany, Paraguay
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Turkish Society of Pediatric Gastroenterology,...CompletedDuchenne Muscular Dystrophy | Pompe Disease (Late-onset)Turkey
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Assy NimerUnknownNon-alcoholic Fatty Liver Disease | Cholesterol Ester Storage DiseaseIsrael
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National Institute of Neurological Disorders and...CompletedGaucher's DiseaseUnited States
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LYSOGENETerminatedGM1 GangliosidosisUnited Kingdom, United States, France
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LYSOGENECasimir, LLC; Cure GM1 FoundationCompleted
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The Hospital for Sick ChildrenCompletedGangliosidoses, GM2 | Tay-Sachs Disease | Sandhoff DiseaseCanada
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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...UnknownGaucher Disease Type 1United States
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University of PennsylvaniaPassage Bio, Inc.RecruitingGM1 GangliosidosisUnited States, Turkey, Brazil, Canada, United Kingdom
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National Center for Research Resources (NCRR)University of PittsburghUnknownGaucher's DiseaseUnited States
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Lysosomal and Rare Disorders Research and Treatment...RecruitingBone Diseases | Gaucher DiseaseUnited States
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Centre Hospitalier Universitaire de NiceCompleted
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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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CENTOGENE GmbH RostockWithdrawnCholesterol Ester Storage Disease | Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Wolman TypeGermany, India, Sri Lanka
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Massachusetts General HospitalActive, not recruiting
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SanofiCompletedGaucher DiseaseUnited States
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Assiut UniversityNot yet recruiting
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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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SanofiCompletedGaucher Disease | SplenomegalyFrance
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The Hospital for Sick ChildrenActelionCompletedGangliosidoses GM2Canada
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Rambam Health Care CampusUnknown
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Massachusetts General HospitalRecruiting
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CENTOGENE GmbH RostockWithdrawnSkeletal Abnormalities | Psychomotor RetardationGermany, India, Sri Lanka, Egypt
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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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SanofiRecruitingGaucher's Disease Type IIIUnited States, Germany, Hungary, France, China, Japan, Canada, Argentina, Italy, Turkey
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Hospices Civils de LyonUnknownLiver Post-transplant PatientsFrance
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Children's Hospital Medical Center, CincinnatiCompletedOsteopenia | Gaucher's DiseaseIsrael, United States
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Assiut UniversityNot yet recruitingPsychiatric Problem | Wolman Disease
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Rigshospitalet, DenmarkUltragenyx Pharmaceutical IncCompletedTarui Disease | Debrancher Deficiency | GYG1 DEFICIENCYDenmark
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University of British ColumbiaCanadian Institutes of Health Research (CIHR); Saudi Arabian Cultural Bureau...Unknown
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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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Azafaros A.G.Active, not recruitingGM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Brazil, France, Germany, Italy, United Kingdom
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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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Children's National Research InstituteActelionCompletedSandhoff Disease | GM2 Gangliosidoses | Tay-SachsUnited States
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Genzyme, a Sanofi CompanyCRL/MedinetTerminatedFabry Disease | Chronic Kidney Disease, Stage IV (Severe)United States
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Hospices Civils de LyonUnknownEnd Stage Renal Disease | Fabry Disease | Renal DialysisFrance
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University of MinnesotaRecruitingInflammation | Oxidative Stress | Gaucher Disease Type IUnited States
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National Institute of Neurological Disorders and...Completed
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Cliniques universitaires Saint-Luc- Université...UnknownGlucose 6 Phosphatase Deficiency | Glycogen Storage Disease Type IBelgium
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Children's Memorial Health Institute, PolandDepartment of Internal Medicine, Hypertension and Vascular Diseases, The...Not yet recruitingGlucose 6 Phosphatase DeficiencyPoland
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Forge Biologics, IncNot yet recruiting