Search clinical trials for: mucopolysaccharidosis
Total 228 results
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Lundquist Institute for Biomedical Innovation at...Not yet recruitingMucopolysaccharidosis III | Sanfilippo SyndromeUnited States
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Denali Therapeutics Inc.RecruitingMucopolysaccharidosis Type IIIAUnited States
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Orchard TherapeuticsRecruitingMPS-IH (Hurler Syndrome)Netherlands, United States, Italy, United Kingdom
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JCR Pharmaceuticals Co., Ltd.RecruitingMucopolysaccharidosis III-AGermany
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SanofiRecruitingOsteochondrodysplasiaAustralia, Spain, Korea, Republic of, China, Italy
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Institut National de la Santé Et de la Recherche...RecruitingMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI | Mucopolysaccharidosis III | Mucopolysaccharidosis IV | Mucopolysaccharidosis VII | Multiple Sulfatase Deficiency Disease | Mucopolysaccharidosis IXFrance
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TakedaRecruitingHunter SyndromeCanada, France
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University of California, San FranciscoRecruiting
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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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Lysosomal and Rare Disorders Research and Treatment...Team SanfilippoRecruitingSanfilippo Syndrome | MPS3United States
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Centre Hospitalier Universitaire de LiegeSanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaboratorsRecruitingCongenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditionsBelgium
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Immusoft of CA, Inc.RecruitingMucopolysaccharidosis IH/S | Mucopolysaccharidosis ISUnited States
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University of ManchesterManchester University NHS Foundation Trust; Great Ormond Street Hospital for... and other collaboratorsRecruiting
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Masonic Cancer Center, University of MinnesotaRecruitingMucopolysaccharidosis Type I | Hematopoietic Cell TransplantationUnited States
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University of California, San FranciscoRecruitingMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI | Wolman Disease | Pompe Disease Infantile-Onset | Mucopolysaccharidosis IV A | Mucopolysaccharidosis VII | Neuronopathic Gaucher DiseaseUnited States
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Sanguine BiosciencesDenali Therapeutics Inc.RecruitingMucopolysaccharidosis III-AUnited States
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TakedaTakeda Development Center Americas, Inc.RecruitingHunter Syndrome | Mucopolysaccharidosis (MPS)United States
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GC Biopharma CorpRecruitingMucopolysaccharidosis II | Hunter SyndromeKorea, Republic of
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Denali Therapeutics Inc.RecruitingMucopolysaccharidosis IIUnited States, Spain, United Kingdom, Czechia, France, Canada, Italy, Belgium, Netherlands, Germany, Argentina, Sweden, Turkey, Colombia
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Manchester University NHS Foundation TrustOrchard TherapeuticsRecruitingMucopolysaccharidosesUnited Kingdom
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Nemours Children's ClinicEunice Kennedy Shriver National Institute of Child Health and Human Development...RecruitingMucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVAUnited States
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AO GENERIUMRecruitingMetabolic Diseases | Mucopolysaccharidosis Type IIRussian Federation
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Children's Hospital of Orange CountyUltragenyx Pharmaceutical IncRecruitingInflammation | Mucopolysaccharidoses | Cardiac Disease | Carotid DiseaseUnited States
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TakedaRecruiting
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Duke UniversityRecruiting
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Mackay Memorial HospitalSanofi Taiwan Co. LtdRecruiting
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Samsung Medical CenterNot yet recruitingSanfilippo Syndrome A
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Shrouk Shaban BakrRecruiting
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SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt)RecruitingGangliosidoses | Galactosialidosis | Sialidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Morquio B Disease | Gm2-Gangliosidosis, Variant B1 | GM2 Activator DeficiencyGermany
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JCR Pharmaceuticals Co., Ltd.RecruitingMucopolysaccharidosis IIUnited States, Germany, Spain, France, Brazil, Argentina, Turkey, United Kingdom, Poland, Italy
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Denali Therapeutics Inc.RecruitingMucopolysaccharidosis IIUnited States, Netherlands, Canada, United Kingdom
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Ultragenyx Pharmaceutical IncPRA Health SciencesRecruitingSly Syndrome | Mucopolysaccharidosis VII | MPS VII | MPS 7United States, Germany, Brazil, Argentina, France, Netherlands, Portugal, Spain, Turkey
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University of PittsburghRecruitingLysosomal Storage Diseases | Leukodystrophy | Niemann-Pick Diseases | Batten Disease | Gaucher Disease | Krabbe Disease | Alpha-Mannosidosis | Osteopetrosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | GM1 Gangliosidoses | MPS I | MPS II | Vanishing White Matter Disease | Multiple Sulfatase Deficiency... and other conditionsUnited States
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GOIZETBioMarin PharmaceuticalRecruiting
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Children's Hospital of PhiladelphiaEli Lilly and Company; University of Pennsylvania; Takeda; National Institute of... and other collaboratorsRecruitingMucopolysaccharidoses | Leukoencephalopathies | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2... and other conditionsUnited States
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Joanne Kurtzberg, MDThe Marcus FoundationRecruitingMucopolysaccharidosis II | Brain Diseases, Metabolic, Inborn | Adrenoleukodystrophy | Batten Disease | Leukodystrophy, Metachromatic | Leukodystrophy, Globoid Cell | Alpha-Mannosidosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | Neimann Pick Disease | Sanfilippo MucopolysaccharidosesUnited States
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Masonic Cancer Center, University of MinnesotaRecruitingHurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Metachromatic Leukodystrophy | Alpha-Mannosidosis | Hunter Syndrome | Mucopolysaccharidosis Disorders | Maroteaux Lamy Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Glycoprotein Metabolic Disorders | Recessive Leukodystrophies | Globoid... and other conditionsUnited States
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St. Jude Children's Research HospitalRecruitingMucopolysaccharidosis I | Severe Combined Immunodeficiency | Neuroblastoma | Neoplasm | Malignancy, HematologicUnited States
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Genzyme, a Sanofi CompanyRecruitingMucopolysaccharidosis I (MPS I)Croatia, United States, Argentina, Australia, Belgium, Brazil, Canada, Denmark, France, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Lebanon, Malaysia, Netherlands, Pakistan, Philippines, Poland, Saudi Arabia, Singapore, Slovaki... and more
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Denali Therapeutics Inc.CompletedMucopolysaccharidosis IIUnited Kingdom, United States, Italy, Netherlands
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Denali Therapeutics Inc.Enrolling by invitationMucopolysaccharidosis IIUnited States, Canada, Belgium, Czechia, Netherlands
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ShireActive, not recruitingHunter SyndromeUnited States, United Kingdom, Canada
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Green Cross CorporationCompleted
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ShireActive, not recruitingHunter SyndromeUnited States, Spain, Canada, United Kingdom, Mexico, Australia, France
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TakedaAvailableHunter SyndromeUnited States, Australia, Mexico, Spain, United Kingdom
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Phoenix NestNational Institute of Neurological Disorders and Stroke (NINDS)Enrolling by invitationSanfilippo Syndrome Type DUnited States
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RTI InternationalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationPrimary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditionsUnited States
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Bellicum PharmaceuticalsTerminatedHemoglobinopathies | Hemophagocytic Lymphohistiocytosis | Metabolic Disorders | Primary Immune Deficiency Disorders | Inherited Bone Marrow Failure SyndromeUnited States
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JCR Pharmaceuticals Co., Ltd.Active, not recruitingMucopolysaccharidosis IUnited States, Brazil, Japan
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JCR Pharmaceuticals Co., Ltd.Active, not recruitingMucopolysaccharidosis IIJapan