Clinical Trials on Mucopolysaccharidosis IV in United States
Total 2391 results
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ShireCompletedMucopolysaccharidosis (MPS)United Kingdom, Netherlands
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University of ChicagoNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedKrabbe Disease | Mucopolysaccharidosis Type II (MPS II) | Mucopolysaccharidosis Type I (MPS I) | Mucopolysaccharidosis Type III (MPS III) | Mucopolysaccharidosis Type VI (MPS VI)United States
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Joyce FoxUltragenyx Pharmaceutical IncNo longer availableMucopolysaccharidosis Type 7United States
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Oregon Health and Science UniversityUniversity of Utah; BioMarin PharmaceuticalCompleted
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Sangamo TherapeuticsTerminated
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Alexion PharmaceuticalsTerminatedMucopolysaccharidosis III, Type B (MPS IIIB) | Sanfilippo BUnited Kingdom
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Lundquist Institute for Biomedical Innovation at...Active, not recruitingMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VIUnited States
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Masonic Cancer Center, University of MinnesotaCompletedMucopolysaccharidosis I | Hurler SyndromeUnited States
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REGENXBIO Inc.Enrolling by invitation
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Orchard TherapeuticsRecruitingMPS-IH (Hurler Syndrome)Netherlands, United States, Italy, United Kingdom
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BioMarin PharmaceuticalCompleted
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Alexion PharmaceuticalsTerminatedMPS IIIB (Sanfilippo B Syndrome)Spain, United States, Brazil, United Kingdom, Portugal, Italy
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Lundquist Institute for Biomedical Innovation at...TerminatedMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VIUnited States
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ArmaGen, IncCompletedMucopolysaccharidosis IUnited States
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Lundquist Institute for Biomedical Innovation at...Not yet recruitingMucopolysaccharidosis III | Sanfilippo SyndromeUnited States
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BioMarin PharmaceuticalCompletedMucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)United States, France, Australia, Germany, Ireland, Austria, Belgium, Italy, Lithuania, Netherlands, Portugal, Sweden, United Kingdom
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Inventiva PharmaCompletedMucopolysaccharidosis VIUnited Kingdom, France, Germany, Portugal
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LYSOGENECompletedMucopolysaccharidosis IIIAGermany, Brazil, United Kingdom, France, Netherlands
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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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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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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Canada
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BioMarin PharmaceuticalCompletedMPS IV AUnited States, Canada, France, United Kingdom, Taiwan, Argentina, Colombia, Japan, Saudi Arabia, Netherlands, Denmark, Korea, Republic of, Brazil, Germany, Portugal, Italy, Qatar
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Ultragenyx Pharmaceutical IncAbeona Therapeutics, IncTerminatedMucopolysaccharidosis III | Sanfilippo Syndrome | MPS IIIA | Sanfilippo AUnited States, Spain, Australia
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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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Ultragenyx Pharmaceutical IncCompleted
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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Brazil
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Ultragenyx Pharmaceutical IncCompletedMucopolysaccharidosis | Sly Syndrome | MPS VII | MPS 7United States
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JCR Pharmaceuticals Co., Ltd.Enrolling by invitationMucopolysaccharidosis IIUnited States
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REGENXBIO Inc.CompletedMucopolysaccharidosis IIBrazil, United States, United Kingdom
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Ultragenyx Pharmaceutical IncCompletedMucopolysaccharidosis | Sly Syndrome | Mucopolysaccharidosis VII | MPS VIISpain, United States, Portugal
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ShireCompletedSanfilippo Syndrome Type BUnited States, United Kingdom, Germany
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BioMarin PharmaceuticalCompletedMucopolysaccharidosis VI | Maroteaux-Lamy SyndromeUnited States, Portugal, France
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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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Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedMucopolysaccharidosis I | Hurler's Syndrome | Hurler-Scheie Syndrome | Scheie's SyndromeUnited States
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Ultragenyx Pharmaceutical IncCompletedMucopolysaccharidosis | Sly Syndrome | Mucopolysaccharidosis VII | MPS VIIUnited States, Brazil, Mexico, Portugal
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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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Denali Therapeutics Inc.Enrolling by invitationMucopolysaccharidosis IIUnited States, Canada, Belgium, Czechia, Netherlands
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Albert Einstein College of MedicineEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationMucopolysaccharidosis II | Fabry Disease | Niemann-Pick Disease, Type C | Mucopolysaccharidosis VI | Lysosomal Acid Lipase Deficiency | Metachromatic Leukodystrophy | Gaucher Disease | Cerebrotendinous Xanthomatosis | Mucopolysaccharidosis IV A | GM1 Gangliosidosis | Mucopolysaccharidosis VII | Acid Sphingomyelinase... and other conditionsUnited States
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Masonic Cancer Center, University of MinnesotaCompletedMucopolysaccharidosis I | Mucopolysaccharidosis VI | Mannosidosis | Mucolipidosis Type II (I-cell Disease)United States
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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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ShireCompletedHunter Syndrome | Mucopolysaccharidosis (MPS)United States, Argentina, Mexico, Spain, United Kingdom
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Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedMucopolysaccharidosis I | Hurler-Scheie Syndrome | Hurlers SyndromeUnited States, Canada, Germany
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Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedMucopolysaccharidosis I | Scheie Syndrome | Hurler Syndrome | Hurler-Scheie SyndromeNetherlands, France, Germany, United Kingdom
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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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Eunice Kennedy Shriver National Institute of Child...University of MinnesotaCompleted
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Denali Therapeutics Inc.RecruitingMucopolysaccharidosis IIUnited States, Spain, United Kingdom, Czechia, France, Canada, Italy, Belgium, Netherlands, Germany, Argentina, Sweden, Turkey, Colombia, Brazil
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REGENXBIO Inc.Enrolling by invitation
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TakedaTakeda Development Center Americas, Inc.RecruitingHunter Syndrome | Mucopolysaccharidosis (MPS)United States
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REGENXBIO Inc.Withdrawn
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Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedMucopolysaccharidosis I | Scheie Syndrome | Hurler's Syndrome | Hurler-Scheie SyndromeUnited States, Canada, United Kingdom, Brazil, Germany, Italy, Netherlands