Search clinical trials for: lysosomal storage diseases
Total 376625 results
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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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Ultragenyx Pharmaceutical IncAbeona Therapeutics, IncEnrolling by invitationMucopolysaccharidosis III-AUnited States, Australia, Spain
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Ultragenyx Pharmaceutical IncAbeona Therapeutics, IncActive, not recruitingMucopolysaccharidosis III | Sanfilippo Syndrome | MPS IIIA | Sanfilippo AUnited States, Australia, Spain
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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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ShireCompletedHunter SyndromeUnited States, United Kingdom, Canada
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ShireCompletedHunter SyndromeUnited States, Spain, Canada, United Kingdom, Mexico, Australia, France
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University of PittsburghNational Institute of Neurological Disorders and Stroke (NINDS)Active, not recruiting
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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
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University Health Network, TorontoOzmosis Research Inc.Completed
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Shoshana VilkGenzyme, a Sanofi CompanyCompletedGaucher Disease | Patient Reported OutcomeIsrael
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Green Cross CorporationCompleted
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Sangamo TherapeuticsEnrolling by invitationFabry Disease | Fabry Disease, Cardiac VariantUnited States, Australia, United Kingdom
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Genzyme, a Sanofi CompanyTerminatedFabry Disease | Alpha Galactosidase A DeficiencyUnited States, Austria, United Kingdom
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4D Molecular TherapeuticsActive, not recruiting
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4D Molecular TherapeuticsActive, not recruitingFabry DiseaseUnited States
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Amicus TherapeuticsActive, not recruitingPompe DiseaseUnited States, Netherlands, United Kingdom, Australia, Germany, New Zealand
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TakedaAvailableHunter SyndromeUnited States, Australia, Mexico, Spain, United Kingdom
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II;Pompe's DiseaseCzechia, Taiwan, Belgium, Germany, Korea, Republic of, United States, Argentina, Australia, Austria, Brazil, Canada, Denmark, France, Hungary, Italy, Japan, Mexico, Netherlands, Poland, Portugal, Russian Federation, Spain, Switzerland, Turk... and more
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Amicus TherapeuticsEngage Health Inc.CompletedFabry DiseaseUnited States
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Phoenix NestNational Institute of Neurological Disorders and Stroke (NINDS)Enrolling by invitationSanfilippo Syndrome Type DUnited States
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TakedaCompletedGaucher DiseaseUnited States
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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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Prevail TherapeuticsEli Lilly and CompanyActive, not recruitingGaucher Disease, Type 2United States, United Kingdom
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Amicus TherapeuticsActive, not recruitingPompe Disease (Late-onset)United States, Germany, Taiwan, Belgium, Australia, Slovenia, Spain, Netherlands, Hungary, Japan, Austria, United Kingdom, France, Argentina, Canada, Sweden, Denmark, New Zealand, Greece, Korea, Republic of, Bosnia and Herzegovina, Ita... and more
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Genzyme, a Sanofi CompanyCompleted
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JCR Pharmaceuticals Co., Ltd.Active, not recruitingMucopolysaccharidosis IUnited States, Brazil, Japan
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JCR Pharmaceuticals Co., Ltd.Active, not recruitingMucopolysaccharidosis IIJapan
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Bio Sidus SACompleted
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Caroline Michaela KistorpRigshospitalet, Denmark; Amicus TherapeuticsActive, not recruiting
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IntraBio IncCompletedGM2 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Germany, Spain, United Kingdom
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Genzyme, a Sanofi CompanyActive, not recruiting
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SanofiCompletedFabry DiseaseUnited Arab Emirates
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BioMarin PharmaceuticalICON plcCompletedMucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVAUnited States, United Kingdom, Australia, Taiwan, Belgium, Malaysia, Austria, Canada, Portugal, France, Ireland, Czechia, Denmark, Italy, Netherlands, Poland, Puerto Rico
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II Pompe DiseaseUnited States, Belgium, Denmark, France, Germany, Netherlands, United Kingdom
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Azafaros A.G.Active, not recruitingNiemann-Pick Disease, Type C | GM2 GangliosidosisBrazil
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TakedaActive, not recruiting