Search clinical trials for: Lysomale Speicherkrankheit
Total 945 results
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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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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
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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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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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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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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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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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National Organization for Rare DisordersFood and Drug Administration (FDA)Active, not recruitingMetachromatic LeukodystrophyUnited States
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LYSOGENETerminatedGM1 GangliosidosisUnited Kingdom, United States, France
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LYSOGENECasimir, LLC; Cure GM1 FoundationCompleted
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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
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Manchester University NHS Foundation TrustNorthern Care Alliance NHS Foundation TrustActive, not recruiting
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Duke UniversityCompleted
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Dr. Anupam SehgalTaysha Gene Therapies, Inc.; GlycoNetActive, not recruitingInfantile GM2 Gangliosidosis (Disorder)Canada
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ShireTerminatedFabry DiseaseSpain, Greece, Germany, Portugal, Canada, Finland, Poland, Sweden