- ICH GCP
- US Clinical Trials Registry
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Search clinical trials for: Lysosomal Storage Disease, Nervous System
Total 246586 results
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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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AVROBIOWithdrawn
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University of FloridaEunice Kennedy Shriver National Institute of Child Health and Human Development...Completed
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Amicus TherapeuticsAvailablePompe Disease Infantile-OnsetTaiwan, United States, Italy
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Amicus TherapeuticsAvailable
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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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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
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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
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Amicus TherapeuticsCompletedFabry DiseaseJapan, Taiwan, Turkey, United States, Brazil
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Chiesi Farmaceutici S.p.A.Active, not recruitingFabry DiseaseUnited States, Netherlands, Australia, Hungary, United Kingdom, Canada, Czechia, Finland, France, Italy, Norway, Slovenia, Spain
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Chiesi Farmaceutici S.p.A.Active, not recruitingFabry DiseaseBelgium, United States, United Kingdom, Italy, Czechia, Denmark, Norway
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PfizerCompleted
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Talaris Therapeutics Inc.Duke UniversityTerminatedNiemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...United States
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Chiesi Farmaceutici S.p.A.Chiesi USA, Inc.AvailableFabry DiseaseUnited States
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Massachusetts General HospitalCompleted
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Spark TherapeuticsCompletedLysosomal Storage Diseases | Glycogen Storage Disease Type 2 | Pompe Disease (Late-onset) | Pompe Disease | LOPD | Acid Maltase DeficiencyUnited States, Italy, United Kingdom, Netherlands, France, Germany
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Genzyme, a Sanofi CompanyCompletedPompe Disease | Glycogen Storage Disease Type II (GSD II) | Acid Maltase DeficiencyUnited States, Belgium, Denmark, France, Germany, Netherlands, United Kingdom
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Centre Hospitalier Universitaire de NiceCompleted
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Chiesi Farmaceutici S.p.A.Iqvia Pty Ltd; ProtalixCompletedFabry DiseaseUnited States, Belgium, Denmark, Italy, United Kingdom
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Lundquist Institute for Biomedical Innovation at...Active, not recruitingMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VIUnited States
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HaEmek Medical Center, IsraelTerminatedLiver Fibrosis | Gaucher Disease | Spleen; FibrosisIsrael
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Mandos LLCCompletedNiemann-Pick Disease, Type CUnited States, Australia, United Kingdom, Germany, France, New Zealand, Singapore, Spain, Turkey
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Shaare Zedek Medical CenterCompletedGaucher Disease, Type 1Israel
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CENTOGENE GmbH RostockWithdrawnSkeletal Abnormalities | Psychomotor RetardationGermany, India, Sri Lanka, Egypt
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CENTOGENE GmbH RostockWithdrawnNiemann-Pick Disease, Type C | Niemann-Pick DiseaseGermany, India, Sri Lanka
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CENTOGENE GmbH RostockWithdrawnSeizures | Mental Retardation | Hepato-splenomegaly | Dysostosis MultiplexEgypt, Germany, India, Sri Lanka