- ICH GCP
- US Clinical Trials Registry
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Search clinical trials for: Peroxisomal storage diseases
Total 376273 results
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Azafaros A.G.Active, not recruitingNiemann-Pick Disease, Type C | GM2 GangliosidosisBrazil
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Rigshospitalet, DenmarkCompletedGlycogen Storage Disease Type IIIDenmark
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TakedaActive, not recruiting
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Rigshospitalet, DenmarkUltragenyx Pharmaceutical IncCompletedTarui Disease | Debrancher Deficiency | GYG1 DEFICIENCYDenmark
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Lundquist Institute for Biomedical Innovation at...Swedish Orphan Biovitrum; Cure Sanfilippo FoundationCompleted
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SanofiActive, not recruitingGaucher's Disease Type III | Gaucher's Disease Type ICanada, Argentina, France, Italy, Japan, Russian Federation, Spain, Sweden, Turkey, United Kingdom
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Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCTerminatedMucopolysaccharidosis I | Hurler's Syndrome | Hurler-Scheie Syndrome | ScheieItaly
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Vitaflo International, LtdUniversity College London Hospitals; University Medical Center Groningen; University... and other collaboratorsCompletedGlycogen Storage DiseaseUnited States, United Kingdom, France, Netherlands
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Masonic Cancer Center, University of MinnesotaTerminatedSickle Cell Disease | Thalassemia | Cerebral Adrenoleukodystrophy | Inherited Metabolic Disorders | High Risk Hematologic DisordersUnited States
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SanofiCompletedPompe Disease | Mucopolysaccharidosis Type I (MPS I)Italy
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University of ManchesterUniversity College, London; Manchester University NHS Foundation Trust; Orchard... and other collaboratorsActive, not recruitingMucopolysaccharidosis Type IIIAUnited Kingdom
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bluebird bioActive, not recruitingCerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)United States, Argentina, Australia, Brazil, France, Germany, Netherlands, United Kingdom, Italy
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AdventHealth Translational Research InstituteTakeda; Sanford-Burnham Medical Research InstituteActive, not recruitingObesity | Lipid Metabolism Disorders | Disorder of Lipid Storage and Metabolism | Metabolic DisorderUnited States
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National Taiwan University HospitalCompletedStress Urinary Incontinence | Urinary Urge Incontinence | Urinary Frequency More Than Once at NightTaiwan
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Recordati Rare DiseasesCompletedCystinosisBelgium, France, United Kingdom, Germany, Italy
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ShireTerminated
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Genzyme, a Sanofi CompanyActive, not recruitingTay-Sachs Disease | Sandhoff DiseaseUnited States, Argentina, Czechia, Portugal, Russian Federation, Turkey, United Kingdom, Brazil, France, Germany, Italy, Japan, Spain
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SanofiCompleted
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TakedaCompleted
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Passage Bio, Inc.SuspendedLeukodystrophy, Globoid CellUnited States, Brazil, Netherlands, United Kingdom, Canada, Israel
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AVROBIOTerminatedGaucher DiseaseUnited States, Canada
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Orchard TherapeuticsOspedale San RaffaeleActive, not recruitingLysosomal Storage Diseases | Metachromatic LeukodystrophyItaly
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TakedaActive, not recruiting
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Mandos LLCTerminatedNiemann-Pick Type C DiseaseUnited States
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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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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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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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Minoryx Therapeutics, S.L.Active, not recruitingAdrenoleukodystrophySpain, United States, Hungary, France, Germany, Italy, Netherlands, United Kingdom
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Chiesi ItaliaCompleted
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Orchard TherapeuticsOspedale San RaffaeleActive, not recruitingLysosomal Storage Disease | Metachromatic LeukodystrophyItaly