Clinical Trials on Inherited Metabolic Storage Diseases in United States
Total 26820 results
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Phoenix NestNational Institute of Neurological Disorders and Stroke (NINDS)Enrolling by invitationSanfilippo Syndrome Type DUnited States
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Masonic Cancer Center, University of MinnesotaTerminatedHurler Syndrome | Mucopolysaccharidosis Type IH | MPS IUnited States
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Alexion PharmaceuticalsCompletedMucopolysaccharidosis IIIBUnited States, United Kingdom, Spain
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Horizon Pharma USA, Inc.CompletedCystinosisFrance, United States, Netherlands
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University of CambridgeSanofiRecruiting
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Nadia Ali, PhDBioMarin PharmaceuticalCompletedMucopolysaccharidosis IV | Morquio DiseaseUnited States
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Masonic Cancer Center, University of MinnesotaCompletedWolman Disease | Adrenoleukodystrophy | Metachromatic Leukodystrophy | Sanfilippo Syndrome | GM1 Gangliosidosis | Globoid Cell Leukodystrophy | Tay Sachs Disease | Sandhoffs Disease | I-Cell DiseaseUnited States
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Idorsia Pharmaceuticals Ltd.CompletedFabry DiseaseUnited States, Spain, Belgium, Australia, Austria, Canada, Germany, Ireland, Italy, Netherlands, Norway, Poland, Switzerland, United Kingdom
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University College, LondonUniversity of Sydney; University Hospital BirminghamUnknownFabry DiseaseUnited Kingdom, Australia
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ShireCompletedSanfilippo Syndrome Type AUnited States
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Masonic Cancer Center, University of MinnesotaCompleted
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AlexionRecruitingWolman Disease | Cholesterol Ester Storage Disease | Lysosomal Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 | Acid Lipase Deficiency | LIPA Deficiency | LAL-DeficiencyFrance, Belgium, United States, Spain, Germany, Greece, Israel, Italy, Slovenia, United Kingdom, Brazil, Canada, Denmark, Australia, Croatia, Czechia, Ireland, Mexico, Netherlands, Poland, Portugal, Saudi Arabia
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Ultragenyx Pharmaceutical IncAbeona Therapeutics, IncEnrolling by invitationMucopolysaccharidosis III-AUnited States, Australia, Spain
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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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ShireTerminatedMetabolic Diseases | Brain Diseases | Central Nervous System Diseases | Nervous System Diseases | Demyelinating Diseases | Genetic Diseases, Inborn | Metabolism, Inborn Errors | Lysosomal Storage Diseases | Lipid Metabolism Disorders | Sphingolipidoses | Hereditary Central Nervous System Demyelinating Diseases and other conditionsUnited States, Denmark, France, Argentina, Belgium, Brazil, Canada, Germany, Japan, Turkey
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Allievex CorporationEnrolling by invitationMPS III BUnited States, Germany, Turkey, United Kingdom, Colombia
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Oregon Health and Science UniversityUniversity of Utah; BioMarin PharmaceuticalCompleted
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Manchester University NHS Foundation TrustOrchard TherapeuticsRecruitingMucopolysaccharidosesUnited Kingdom
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseAustralia, Netherlands, United Kingdom, Canada, Czechia, Norway, Slovenia
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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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Phoenix NestAparito Ltd.; Vaincres Les Maladies Lysosomales; Association Sanfilippo SudNot yet recruitingSanfilippo Syndrome Type CUnited States, France
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Children's Hospital of Orange CountyUltragenyx Pharmaceutical IncRecruitingInflammation | Mucopolysaccharidoses | Cardiac Disease | Carotid DiseaseUnited States
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ArmaGen, IncCompletedMucopolysaccharidosis IUnited States
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CENTOGENE GmbH RostockCompletedFabry Disease | Anderson-Fabry Disease | Fabry´s DiseaseArgentina, Belgium, Croatia, Czechia, Denmark, France, Germany, United Kingdom
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National Institute of Allergy and Infectious Diseases...CompletedInherited Immune DeficienciesUnited States
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BioMarin PharmaceuticalCompletedMPS VIUnited States, Australia, France, Brazil, Germany, Portugal, United Kingdom
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Genzyme, a Sanofi CompanyCompletedFabry DiseaseUnited States, United Kingdom, Canada, Hungary, Poland, Czech Republic
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Emory UniversityBioMarin PharmaceuticalCompletedMPS - Mucopolysaccharidosis | Morquio DiseaseUnited States
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Zymenex A/SEuropean CommissionCompletedAlpha-MannosidosisBelgium, Denmark, Spain, United Kingdom
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ShireCompletedSanfilippo Syndrome Type BUnited States, United Kingdom, Germany
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Enzyvant Therapeutics GmBHCompletedFarber Disease | Farber's Disease | Farber Lipogranulomatosis | Acid Ceramidase Deficiency | Ceramidase Deficiency | N-Laurylsphingosine Deacylase Deficiency | ASAH1 MutationUnited States, Egypt, Canada, Italy, Turkey, Argentina, Germany, India, Sweden
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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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University of NebraskaNational Cancer Institute (NCI); VA Nebraska Western Iowa Health Care System; Virginia Mason Hospital/Medical CenterRecruitingDiabetes Mellitus, Type 2 | PreDiabetes | Genetic Predisposition to Disease | Chronic Pancreatitis | Inherited Disease | Pancreas CystUnited States
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Manchester Royal Eye HospitalBioMarin PharmaceuticalUnknownMucopolysaccharidosesUnited Kingdom
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Genzyme, a Sanofi CompanyCompletedGaucher Disease, Type 1 | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency Disease | Gaucher Disease, Non-Neuronopathic FormUnited States, United Kingdom, Spain, Italy, Brazil, Canada, Poland
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National Institute of Neurological Disorders and...CompletedBrain Neoplasm | Alzheimer's Disease | Niemann Pick DiseaseUnited States
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Children's Hospitals and Clinics of MinnesotaBioMarin Pharmaceutical; Gillette Children's Specialty Healthcare; Greenwood...CompletedMucopolysaccharidosis VI | Mucopolysaccharidosis IV AUnited States
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Eunice Kennedy Shriver National Institute of Child...TerminatedNiemann-Pick Disease, Type C1United States
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Vtesse, LLC, a Mallinckrodt Pharmaceuticals CompanyCompletedNiemann-Pick Disease, Type C1United States
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Cyclo Therapeutics, Inc.Active, not recruitingNiemann-Pick Disease, Type C1United States
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SanofiRecruitingFabry DiseaseItaly, United States, Austria, China, Czechia, Denmark, France, Germany, Greece, Japan, Korea, Republic of, Netherlands, Norway, Spain, Taiwan, Turkey, United Kingdom, Canada, Poland
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PfizerCompletedInherited ErythromelalgiaUnited States
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Mayo ClinicNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedNiemann-Pick Disease, Type CUnited States
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Eunice Kennedy Shriver National Institute of Child...Washington University School of Medicine; University of Oxford; National Human...CompletedNiemann-Pick Disease, Type CUnited States
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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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LYSOGENEActive, not recruitingMucopolysaccharidosis Type IIIAGermany, United States, United Kingdom, France, Netherlands
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Ultragenyx Pharmaceutical IncCompletedMucopolysaccharidosis | Sly Syndrome | MPS VII | MPS 7United States
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Mandos LLCTerminatedNiemann-Pick Type C DiseaseUnited States
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Eunice Kennedy Shriver National Institute of Child...Washington University School of Medicine; Weill Medical College of Cornell...CompletedNeimann-Pick DiseaseUnited States
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BioMarin PharmaceuticalCompletedMucopolysaccharidosis VI | Maroteaux-Lamy SyndromeUnited States, Portugal, France