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Search clinical trials for: storage endotheliopathy
Total 1217 results
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Hadassah Medical OrganizationCompleted
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ShireCompleted
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University of FloridaLive Like Bella Pediatric Cancer ResearchCompletedCommon Variable Immunodeficiency | Severe Combined Immunodeficiency | Sickle Cell Disease | Hurler Syndrome | Chronic Granulomatous Disease | Thalassemia | Wiskott-Aldrich Syndrome | Adrenoleukodystrophy | X-linked Lymphoproliferative Disease | Diamond Blackfan Anemia | Bone Marrow Failure Syndrome | Hemophagocytic... and other conditionsUnited States
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseAustralia, Netherlands, United Kingdom, Canada, Czechia, Norway, Slovenia
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Centre Hospitalier Universitaire VaudoisUniversity Hospital, GenevaCompletedChronic Renal Failure | Healthy ControlsSwitzerland
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Denmark, Belgium, United Kingdom, Norway, Italy, Czechia
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IntraBio IncActive, not recruitingNiemann-Pick Disease, Type CUnited States, Australia, United Kingdom, Germany, Slovakia, Netherlands, Czechia, Switzerland
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Greenwood Genetic CenterCompletedGalactosialidosis | Sialidosis | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Beta Mannosidosis | Mucolipidosis II | Mucolipidosis III | Schindler DiseaseUnited States
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Spain, United Kingdom, Paraguay
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Netherlands, Hungary, United Kingdom, Czechia, Norway, Slovenia, Spain, Finland, France, Italy, Switzerland
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Serbia, Spain, United Kingdom, Australia, Paraguay
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Amicus TherapeuticsCompletedPompe Disease (Late-onset)United States, Australia, Korea, Republic of, Japan, Taiwan, Belgium, Slovenia, New Zealand, United Kingdom, France, Argentina, Canada, Spain, Bosnia and Herzegovina, Germany, Sweden, Hungary, Austria, Bulgaria, Denmark, Greece, I... and more
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CENTOGENE GmbH RostockActive, not recruiting
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Cystinose StiftungLabor Becker & Kollegen, Munich, Germany; Screening Labor Hannover; Genetikum... and other collaboratorsCompletedSpinal Muscular Atrophy | Cystinosis | Cystinosis, NephropathicGermany
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Manchester University NHS Foundation TrustCompletedMucopolysaccharidosis Type IIUnited Kingdom
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AVROBIOTerminatedFabry DiseaseAustralia, Brazil
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Mandos LLCTerminatedNiemann-Pick Disease, Type CUnited States, Australia, United Kingdom, Turkey, Switzerland, Germany, New Zealand, Singapore, France
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AVROBIOTerminatedType 1 Gaucher DiseaseUnited States, Canada
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Homology Medicines, IncWithdrawnMucopolysaccharidosis IIUnited States, Canada
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SanofiCompletedSphingomyelin LipidosisItaly, Czechia, Belgium, France, Portugal, Turkey, United States, Argentina, Brazil, Chile, Germany, Romania, Spain
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University Hospital, BordeauxCompletedChronic Pain | Fabry's DiseaseFrance
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Stanford UniversityCompletedObesity | Insulin ResistanceUnited States
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Allievex CorporationEnrolling by invitationMPS III BUnited States, Germany, Turkey, United Kingdom, Colombia
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AVROBIOWithdrawn
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University of FloridaEunice Kennedy Shriver National Institute of Child Health and Human Development...Completed
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Chiesi Farmaceutici S.p.A.Completed
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Chiesi Farmaceutici S.p.A.CompletedAlpha-MannosidosisFrance
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Amicus TherapeuticsAvailablePompe Disease Infantile-OnsetTaiwan, United States, Italy
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Amicus TherapeuticsAvailable
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Ultragenyx Pharmaceutical IncAbeona Therapeutics, IncTerminatedMucopolysaccharidosis III | Sanfilippo Syndrome | MPS IIIA | Sanfilippo AUnited States, Spain, Australia
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Azienda Ospedaliera Universitaria Integrata VeronaCompleted
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ShireTakeda Development Center Americas, Inc.Active, not recruitingMetachromatic Leukodystrophy (MLD)Denmark, Germany, Japan, Australia, France, Brazil, Czechia
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TakedaTakeda Development Center Americas, Inc.Active, not recruitingHunter SyndromeUnited States, Germany, Malaysia, Philippines, Thailand, Dominican Republic, Serbia, Vietnam
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Mansoura UniversityActive, not recruitingLower Urinary Tract Symptoms | Erectile DysfunctionEgypt
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TakedaCompleted
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Genzyme, a Sanofi CompanyActive, not recruitingGlycogen Storage Disease Type II-Pompe's DiseaseUnited States, France, Japan, Taiwan, United Kingdom
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Asklepios Biopharmaceutical, Inc.Active, not recruiting
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JCR Pharmaceuticals Co., Ltd.Active, not recruitingMucopolysaccharidosis IIBrazil
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Mansoura UniversityAhmed Mohamed ElshalCompletedOveractive Bladder | Urinary Incontinence, Urge | Urgency-frequency Syndrome | Post ProstatectomyEgypt
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National Organization for Rare DisordersFood and Drug Administration (FDA)Active, not recruitingMetachromatic LeukodystrophyUnited States
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Southern Illinois UniversityAstellas Scientific & Medical Affairs, Inc.; Sisters of the Third Order of...TerminatedPain | Urinary Bladder, Overactive | Lower Urinary Tract SymptomsUnited States
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Ultragenyx Pharmaceutical IncTerminatedWilson Disease | Ornithine Transcarbamylase Deficiency | Glycogen Storage Disease Type IASpain, United Kingdom, Canada, United States, Brazil
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LYSOGENETerminatedGM1 GangliosidosisUnited Kingdom, United States, France
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LYSOGENECasimir, LLC; Cure GM1 FoundationCompleted
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University of PittsburghActive, not recruitingMetachromatic LeukodystrophyUnited 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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ShireCompletedGaucher DiseaseIndia
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Freeline TherapeuticsTerminatedLysosomal Storage Diseases | Fabry DiseaseUnited States, Norway, Germany, Austria, United Kingdom, Italy, Canada
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Albert Einstein College of MedicineEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationMucopolysaccharidosis II | Fabry Disease | Niemann-Pick Disease, Type C | Mucopolysaccharidosis VI | Lysosomal Acid Lipase Deficiency | Metachromatic Leukodystrophy | Gaucher Disease | Cerebrotendinous Xanthomatosis | Mucopolysaccharidosis IV A | GM1 Gangliosidosis | Mucopolysaccharidosis VII | Acid Sphingomyelinase... and other conditionsUnited States