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Clinical Trials on Inherited Metabolic Storage Diseases in United States
Total 26820 results
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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease, Type CUnited States
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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease, Type CUnited States
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National Niemann-Pick Disease FoundationIcahn School of Medicine at Mount Sinai; Engage Health Inc.CompletedNiemann-Pick DiseasesUnited States
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Genzyme, a Sanofi CompanyTerminatedNiemann-Pick Disease | Acid Sphingomyelinase DeficiencyUnited States
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Janssen Research & Development, LLCRecruitingInherited Retinal DiseasesUnited States, Korea, Republic of, Canada, Spain, Denmark, Germany, France, Italy, Israel, Austria, United Kingdom
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Zymenex A/SEuropean CommissionCompletedA Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-MannosidosisAlpha-MannosidosisDenmark, United Kingdom, France, Germany, Poland
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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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ShireTerminatedSanfilippo Syndrome | Mucopolysaccharidosis (MPS)United States, United Kingdom, Spain, Germany, France, Italy, Netherlands
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CENTOGENE GmbH RostockShire Human Genetic Therapies, Inc.CompletedCerebrovascular AccidentAustria, Belgium, Croatia, Finland, France, Georgia, Germany, Ireland, Italy, Malta, Poland, Portugal, Spain, United Kingdom
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National Institute of Neurological Disorders and...RecruitingAmyotrophic Lateral Sclerosis Type 4 | Inherited Neurological Disorders of RNA ProcessingUnited States
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Allievex CorporationCompletedMucopolysaccharidosis Type IIIB | MPS III BUnited States, Taiwan, Spain, Colombia, Germany, United Kingdom, Turkey
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SanofiCompletedSphingomyelin LipidosisItaly, Czechia, Belgium, France, Portugal, Turkey, United States, Argentina, Brazil, Chile, Germany, Romania, Spain
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Nadia Ali, PhDBioMarin PharmaceuticalCompletedMorquio A Syndrome | Mucopolysaccharidosis IV AUnited States
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Denali Therapeutics Inc.RecruitingMucopolysaccharidosis Type IIIAUnited States
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Ultragenyx Pharmaceutical IncAbeona Therapeutics, IncTerminatedMucopolysaccharidosis III | Sanfilippo Syndrome | MPS IIIA | Sanfilippo AUnited States, Spain, Australia
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)RecruitingBiochemically Recurrent Prostate Carcinoma | Stage IV Prostate Cancer AJCC v8 | Stage IVA Prostate Cancer AJCC v8 | Stage IVB Prostate Cancer AJCC v8 | Metastatic Prostate AdenocarcinomaUnited States
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Patricia I. Dickson, M.D.University of California, Los Angeles; FDA Office of Orphan Products Development and other collaboratorsTerminatedMucopolysaccharidosis I | Lysosomal Storage Diseases | Spinal Cord CompressionUnited States, Finland
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Alexion PharmaceuticalsCompletedMPS IIIB (Sanfilippo Syndrome)Spain, Netherlands, United Kingdom, United States, Brazil
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Massachusetts General HospitalCompletedFabry Disease | Mitochondrial Disease | Small Fiber Neuropathy | Ehlers Danlos SyndromeUnited States
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Allievex CorporationActive, not recruitingMucopolysaccharidosis Type IIIB | MPS III BGermany, United States, Turkey, United Kingdom, Colombia, Spain, Taiwan
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Cyclo Therapeutics, Inc.CompletedNiemann-Pick Disease, Type C1United States
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Genzyme, a Sanofi CompanyRecruitingFabry DiseaseCroatia, United States, Argentina, Australia, Belgium, Bulgaria, Canada, Germany, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Malaysia, Philippines, Poland, Romania, Singapore, Slovakia, Taiwan, Thailand, United Kingdom, Vietn... and more
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Abeona Therapeutics, IncTerminatedMucopolysaccharidosis Type 3 BUnited States, Germany, France, Spain
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Swedish Orphan BiovitrumCompletedSanfilippo Syndrome Type A (MPS IIIA)United States, Turkey
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Ultragenyx Pharmaceutical IncCompletedMucopolysaccharidosis | Sly Syndrome | Mucopolysaccharidosis VII | MPS VIISpain, United States, Portugal
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Inventiva PharmaCompletedMucopolysaccharidosis VIUnited Kingdom, France, Germany, Portugal
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National Eye Institute (NEI)TerminatedInherited Retinal Degeneration | Inherited Ophthalmic DiseasesUnited States
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Fresenius Medical Care North AmericaCompleted
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Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedMucopolysaccharidosis I | Hurler's Syndrome | Hurler-Scheie Syndrome | Scheie's SyndromeUnited States
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Genzyme, a Sanofi CompanyRecruitingGaucher Disease | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency DiseaseHungary, Croatia, United States, Australia, Belgium, Brazil, Bulgaria, Canada, Denmark, Germany, Greece, Hong Kong, India, Indonesia, Italy, Jordan, Korea, Republic of, Kuwait, Lebanon, Malaysia, Pakistan, Philippines, Poland, Saudi Arabia and more
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Ultragenyx Pharmaceutical IncAbeona Therapeutics, IncActive, not recruitingMucopolysaccharidosis III | Sanfilippo Syndrome | MPS IIIA | Sanfilippo AUnited States, Australia, Spain
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ArdelyxCompletedEnd Stage Renal Disease | HyperphosphatemiaUnited States
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedHurler Syndrome | Sanfilippo Syndrome Type A | Sanfilippo Syndrome Type BUnited States
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Amicus TherapeuticsRecruitingFabry DiseaseSpain, United States, Japan, Australia, France, Portugal
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Bellicum PharmaceuticalsTerminatedHemoglobinopathies | Hemophagocytic Lymphohistiocytosis | Metabolic Disorders | Primary Immune Deficiency Disorders | Inherited Bone Marrow Failure SyndromeUnited States
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LYSOGENECompletedMucopolysaccharidosis IIIAGermany, Brazil, United Kingdom, France, Netherlands
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Ultragenyx Pharmaceutical IncCompletedMucopolysaccharidosis | Sly Syndrome | Mucopolysaccharidosis VII | MPS VIIUnited States, Brazil, Mexico, Portugal
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Washington University School of MedicineEunice Kennedy Shriver National Institute of Child Health and Human Development...RecruitingNiemann-Pick Disease, Type CUnited States
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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease Type C1 | Juvenile Neuronal Ceroid Lipofuscinosis | Smith-Lemli-Opitz Syndrome | Creatine Transporter DeficiencyUnited States
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Masonic Cancer Center, University of MinnesotaCompletedNiemann-Pick Disease | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | GM1 Gangliosidosis | Fucosidosis | Globoid Cell Leukodystrophy | Gaucher's Disease | Tay Sachs Disease | Sandhoff DiseaseUnited States
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University of California, San DiegoRaptor Pharmaceuticals Corp.CompletedCystinosis | Nephropathic CystinosisUnited States
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Swedish Orphan BiovitrumCompletedSanfilippo Syndrome Type A (MPS IIIA)United States, Germany, Turkey
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Masonic Cancer Center, University of MinnesotaTerminatedSphingolipidoses | Niemann-Pick Disease, Type C | Krabbe Disease | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Hurler's Syndrome | Maroteaux-Lamy Syndrome | Wolman's Disease | Niemann-Pick Disease Type BUnited States
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BioMarin PharmaceuticalCompletedMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVATaiwan, United States, Italy, United Kingdom
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Orchard TherapeuticsRecruitingMPS-IH (Hurler Syndrome)Netherlands, United States, Italy, United Kingdom
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Children's Hospital Los AngelesCompletedGranuloma | Anemia | Thrombocytopenia | Neutropenia | Sickle Cell Disease | Thalassemia | Niemann-Pick Disease | Wiskott-Aldrich Syndrome | Osteopetrosis | Fucosidosis | Chediak Higashi Syndrome | Hurler DiseaseUnited States
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National Eye Institute (NEI)CompletedHealthy Volunteer | Retinal Disease | Inherited Retinal DegenerationUnited States
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SanofiRecruitingFabry DiseaseChina, Finland, Germany, Norway, United Kingdom, United States, Denmark, Austria, Greece, Romania, Argentina, Brazil, Canada, Japan, Mexico, Poland, Italy, France, Turkey, Australia, Switzerland
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Vanderbilt UniversityCompletedEnd Stage Renal DiseaseUnited States
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State University of New York - Downstate Medical...TerminatedDiabetes Mellitus | End-Stage Renal DiseaseUnited States