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Clinical Trials on Inherited Metabolic Storage Diseases in United States
Total 26820 results
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Genzyme, a Sanofi CompanyTerminatedFabry DiseaseAustria, Brazil, France, United States, Argentina, Belgium, Canada, Finland, Hungary, Norway, Poland, Spain, Taiwan, United Kingdom
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Amicus TherapeuticsCompletedFabry DiseaseFrance, Austria, United States, Belgium, Japan, United Kingdom, Denmark, Brazil, Australia, Italy
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Genzyme, a Sanofi CompanyCompletedSphingomyelin LipidosisUnited States, Belgium, Brazil, France, Germany, Italy, United Kingdom
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Freeline TherapeuticsCompletedLysosomal Storage Diseases | Fabry DiseaseGermany, United Kingdom
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Genzyme, a Sanofi CompanyCompletedFabry DiseaseUnited States, France, Netherlands, Puerto Rico, United Kingdom
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Masonic Cancer Center, University of MinnesotaBioMarin PharmaceuticalTerminated
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Masonic Cancer Center, University of MinnesotaCompletedMucopolysaccharidosis I | Hurler SyndromeUnited States
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Rocket Pharmaceuticals Inc.Active, not recruitingDanon DiseaseUnited States
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SanofiCompletedFabry DiseaseUnited Arab Emirates
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Alexion PharmaceuticalsCompletedWolman Disease | Lysosomal Acid Lipase DeficiencyUnited Kingdom, France, United States, Egypt, Ireland
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Immusoft of CA, Inc.RecruitingMucopolysaccharidosis IH/S | Mucopolysaccharidosis ISUnited States
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Patricia I. Dickson, M.D.The Ryan FoundationTerminatedMucopolysaccharidosis I | Scheie Syndrome | Lysosomal Storage Disease | Spinal Cord Compression | Hurler-Scheie SyndromeFinland, United States
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ShireCompletedFabry DiseaseCanada, United States
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedMucopolysaccharidosesUnited States
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JCR Pharmaceuticals Co., Ltd.Active, not recruitingMucopolysaccharidosis IUnited States, Brazil, Japan
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Sangamo TherapeuticsActive, not recruitingHemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis IIUnited States
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Amicus TherapeuticsTerminatedFabry DiseaseAustralia, United States, Italy, Spain, Belgium, Turkey, Brazil, Argentina, United Kingdom, Denmark, France, Canada, Egypt
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Abeona Therapeutics, IncTerminatedMucopolysaccharidosis III-BUnited States, Germany, France
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Amicus TherapeuticsCompletedFabry DiseaseFrance, United Kingdom
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University of Alabama at BirminghamCompletedProteinuria | Fabry DiseaseUnited States, Slovenia
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedMucopolysaccharidosis Type IUnited States
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Amicus TherapeuticsCompletedOpen-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry DiseaseFabry DiseaseUnited States, Austria, United Kingdom, Argentina, Australia, Belgium, Brazil, Canada, Denmark, Egypt, France, Italy, Japan, Spain, Turkey
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Recordati Rare DiseasesCompletedCystinosisBelgium, France, United Kingdom, Germany, Italy
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SanofiCompletedOsteochondrodysplasiaUnited States
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PTC TherapeuticsEnrolling by invitationInherited Mitochondrial DiseaseUnited States, France, Spain, United Kingdom, Italy, Poland, Japan
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Horizon Pharma USA, Inc.CompletedCystinosisFrance, United States, Netherlands
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Lundquist Institute for Biomedical Innovation at...National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedMucopolysaccharidosesUnited States
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Manchester University NHS Foundation TrustRecruiting
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JCR Pharmaceuticals Co., Ltd.CompletedMucopolysaccharidosis IUnited States, Brazil, Japan
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Memorial Sloan Kettering Cancer CenterRecruitingHemoglobinopathies | AML | MDS | Bone Marrow Failure | ALL | Inherited Metabolic Disorders | HLH | MPD Withou Myelofibrosis | NHL or HLUnited States
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Imperial College LondonRoyal Brompton & Harefield NHS Foundation Trust; Great Ormond Street Hospital...RecruitingCardiovascular Diseases | Inherited Cardiac ConditionsUnited Kingdom
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Joyce FoxUltragenyx Pharmaceutical IncNo longer availableMucopolysaccharidosis Type 7United States
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Sanguine BiosciencesDenali Therapeutics Inc.RecruitingMucopolysaccharidosis III-AUnited States
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Emory UniversityBioMarin PharmaceuticalTerminatedMucopolysaccharidosis Type IVAUnited States
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SanofiPulse Infoframe Ltd.RecruitingNiemann-Pick Diseases | Acid Sphingomyelinase DeficiencyUnited States
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Amicus TherapeuticsCompletedFabry DiseaseUnited States, Italy, Spain, Turkey, Brazil, Canada, United Kingdom, France, Poland, Australia, Egypt, Argentina, Denmark
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Masonic Cancer Center, University of MinnesotaRecruitingMucopolysaccharidosis Type I | Hematopoietic Cell TransplantationUnited States
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Genzyme, a Sanofi CompanyCompletedFabry DiseaseUnited States, United Kingdom, Canada, Czech Republic, Hungary, Poland
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Alice BertainaCalifornia Institute for Regenerative Medicine (CIRM)RecruitingCKD Stage 4 | Cystinosis | FSGS | SLE Nephritis | SIODUnited States
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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, Israel, Mexico, Russian Federation, Argentina, Italy
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REGENXBIO Inc.Enrolling by invitation
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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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Johns Hopkins UniversityNational Human Genome Research Institute (NHGRI)Enrolling by invitationGenetic Counseling | Inherited Cardiac DiseaseUnited States
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Idorsia Pharmaceuticals Ltd.CompletedGM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Gaucher Disease, Type 2 | AB Variant Gangliosidosis GM2United States, Spain, Germany, Italy, Belgium, Brazil, France, Portugal, Switzerland, United Kingdom
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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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Horizon Pharma USA, Inc.CompletedCystinosisFrance, United States, United Kingdom, Belgium, Italy, Netherlands
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ShireNo longer available
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Vanderbilt UniversityNational Heart, Lung, and Blood Institute (NHLBI); University of South Florida and other collaboratorsCompletedHermansky Pudlak SyndromeUnited States
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Alexion PharmaceuticalsCompletedMucopolysaccharidosis IIIBUnited States, United Kingdom, Spain
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Phoenix NestNational Institute of Neurological Disorders and Stroke (NINDS)Enrolling by invitationSanfilippo Syndrome Type DUnited States