Search clinical trials for: storage endotheliopathy
Total 1217 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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Amicus TherapeuticsActive, not recruitingFabry DiseaseUnited States, United Kingdom
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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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TakedaWithdrawnMetachromatic Leukodystrophy (MLD)Spain
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Idorsia Pharmaceuticals Ltd.Active, not recruitingFabry DiseaseUnited States, United Kingdom, Norway, Germany, Australia, Canada, Spain, Switzerland, Austria, Belgium, France, Netherlands, Poland
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SanofiCompletedGaucher's DiseaseUnited States
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SanofiCompletedOsteochondrodysplasiaUnited States
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AVROBIOTerminatedFabry DiseaseUnited States, Australia, Brazil
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SanofiCompletedGaucher's DiseaseUnited States
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University of California, San DiegoEnrolling by invitationCystinosisUnited States
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Icahn School of Medicine at Mount SinaiNational Institute on Deafness and Other Communication Disorders (NIDCD)CompletedMal de Debarquement Syndrome (MdDS)United States
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Mandos LLCTerminatedNiemann-Pick Disease, Type CCosta Rica
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TakedaCompleted
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University of MichiganManiilaq AssociationActive, not recruitingSuicide | Accidental Injuries | Firearm InjuryUnited States
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JCR Pharmaceuticals Co., Ltd.Enrolling by invitationMucopolysaccharidosis IIUnited States
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S.LAB (SOLOWAYS)Center of New Medical Technologies; Triangel ScientificCompletedLDL Hyperlipoproteinemia | Low-Density-Lipoprotein-Type [LDL] Hyperlipoproteinemia | Triglyceride Storage Type I or II DiseaseRussian Federation
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Terence FlotteMassachusetts General Hospital; University of Massachusetts, WorcesterActive, not recruitingTay-Sachs Disease | Sandhoff DiseaseUnited States
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Queen's University, BelfastBelfast Health and Social Care TrustCompletedCOVID | Corona Virus Infection | Multisystem Inflammatory Syndrome in ChildrenUnited Kingdom
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Freeline TherapeuticsCompletedLysosomal Storage Diseases | Fabry DiseaseGermany, United Kingdom
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REGENXBIO Inc.Active, not recruitingHurler Syndrome | Hurler-Scheie Syndrome | Mucopolysaccharidosis Type I (MPS I)United States, Brazil, Israel
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University Children's Hospital, ZurichSanofiCompleted
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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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XVIVO PerfusionActive, not recruitingHeart TransplantationUnited Kingdom, Germany, Austria, Belgium, France, Italy, Spain, Sweden
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Chiesi ItaliaCompleted
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Orchard TherapeuticsOspedale San RaffaeleActive, not recruitingLysosomal Storage Disease | Metachromatic LeukodystrophyItaly
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IntraBio IncCompletedNiemann-Pick Disease, Type CUnited States, United Kingdom, Germany, Slovakia, Spain
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Stanford UniversityNational Cancer Institute (NCI); National Institutes of Health (NIH)Enrolling by invitationLymphoma | Hematologic Diseases | LeukemiaUnited States
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SanofiCompleted
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Rocket Pharmaceuticals Inc.Completed
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)CompletedMalignant Neoplasm | CaregiverUnited States
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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Brazil
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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Canada
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Resverlogix CorpWithdrawn
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ZevraDenmarkAvailableNiemann-Pick Disease, Type CUnited States
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National and Kapodistrian University of AthensCompletedFabry DiseaseGreece
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Spark TherapeuticsActive, not recruitingLysosomal Storage Diseases | Glycogen Storage Disease Type II | Glycogen Storage Disease Type 2 | Pompe Disease (Late-onset) | Pompe Disease | LOPD | Acid Maltase DeficiencyUnited States, Canada, Netherlands, France, Denmark, Germany, Italy, United Kingdom
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agnes chenUniversity of California, Los Angeles; National Institute of Neurological Disorders... and other collaboratorsTerminatedMucopolysaccharidosis I | Cognitive DeclineUnited States
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Duke UniversityGenzyme, a Sanofi CompanyActive, not recruitingPompe Disease (Late-onset) | Pompe Disease | GAA DeficiencyUnited States
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REGENXBIO Inc.Enrolling by invitation
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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 MinnesotaActive, not recruiting
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Instituto de Investigacion Sanitaria La FeTerminated
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Johns Hopkins UniversityCompletedLung Transplant Recipients
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Ultragenyx Pharmaceutical IncTerminatedGlycogen Storage Disease Type IIIUnited States, Netherlands
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Genzyme, a Sanofi CompanyCompleted
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Genzyme, a Sanofi CompanyCompleted
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Genzyme, a Sanofi CompanyActive, not recruitingGlycogen Storage Disease Type IIFrance
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Hospital de Clinicas de Porto AlegreConselho Nacional de Desenvolvimento Científico e Tecnológico; Fundação Médica...CompletedGlycogen Storage Disease Type IBrazil
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Genzyme, a Sanofi CompanyCompletedSphingomyelin LipidosisUnited States, Belgium, Brazil, France, Germany, Italy, United Kingdom