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Clinical Trials on Mucopolysaccharidosis I in United States
Total 1277 results
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Allievex CorporationActive, not recruitingMucopolysaccharidosis Type IIIBUnited States, Germany, Spain, Argentina, Australia, Brazil, Colombia, Taiwan, Turkey
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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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Allievex CorporationActive, not recruitingMucopolysaccharidosis Type IIIB | MPS III BGermany, United States, Turkey, United Kingdom, Colombia, Spain, Taiwan
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Alexion PharmaceuticalsCompletedMPS IIIB (Sanfilippo Syndrome)Spain, Netherlands, United Kingdom, United States, Brazil
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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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ShireCompletedMucopolysaccharidosis (MPS)United Kingdom, Netherlands
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Joyce FoxUltragenyx Pharmaceutical IncNo longer availableMucopolysaccharidosis Type 7United States
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Alexion PharmaceuticalsTerminatedMucopolysaccharidosis III, Type B (MPS IIIB) | Sanfilippo BUnited Kingdom
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Nadia Ali, PhDBioMarin PharmaceuticalCompletedMorquio A Syndrome | Mucopolysaccharidosis IV AUnited States
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BioMarin PharmaceuticalCompletedMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVATaiwan, United States, Italy, United Kingdom
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BioMarin PharmaceuticalTerminatedMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVAUnited Kingdom, United States, Germany
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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...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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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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BioMarin PharmaceuticalTerminatedMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVAUnited States, United Kingdom, Canada, Germany
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LYSOGENECompletedMucopolysaccharidosis IIIAGermany, Brazil, United Kingdom, France, Netherlands
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Sangamo TherapeuticsActive, not recruitingHemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis IIUnited States
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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Canada
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BioMarin PharmaceuticalTerminatedMorquio A Syndrome | MPS IV A | Mucopolysaccharidosis IVAUnited Kingdom
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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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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Brazil
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Ultragenyx Pharmaceutical IncCompleted
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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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BioMarin PharmaceuticalApproved for marketingMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVAUnited States, Puerto Rico
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Nadia Ali, PhDBioMarin PharmaceuticalCompletedMucopolysaccharidosis IV | Morquio DiseaseUnited 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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BioMarin PharmaceuticalCompletedMucopolysaccharidosis VI | Maroteaux-Lamy SyndromeUnited States, Portugal, France
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ShireCompletedSanfilippo Syndrome Type BUnited States, United Kingdom, Germany
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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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Ultragenyx Pharmaceutical IncCompletedMucopolysaccharidosis | Sly Syndrome | Mucopolysaccharidosis VII | MPS VIIUnited States, Brazil, Mexico, Portugal
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BioMarin PharmaceuticalTerminatedMorquio A Syndrome | MPS IV A | Mucopolysaccharidosis IVAFrance, United Kingdom, Taiwan, United States, Argentina, Netherlands, Canada, Brazil, Germany, Italy
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BioMarin PharmaceuticalICON plcCompletedMucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVAUnited States, United Kingdom, Australia, Taiwan, Belgium, Malaysia, Austria, Canada, Portugal, France, Ireland, Czechia, Denmark, Italy, Netherlands, Poland, Puerto Rico
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BioMarin PharmaceuticalCompletedMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IV AUnited States, Canada, France, Taiwan, Argentina, Colombia, Spain, Turkey, Japan, Saudi Arabia, Netherlands, Denmark, Korea, Republic of, Brazil, United Kingdom, Germany, Norway, Portugal, Italy, Qatar
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Denali Therapeutics Inc.Enrolling by invitationMucopolysaccharidosis IIUnited States, Canada, Belgium, Czechia, Netherlands
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Birmingham Women's and Children's NHS Foundation...BioMarin PharmaceuticalWithdrawnMucopolysaccharidosis IV | Morquio A DiseaseUnited 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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ShireCompletedHunter Syndrome | Mucopolysaccharidosis (MPS)United States, Argentina, Mexico, Spain, United Kingdom
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Masonic Cancer Center, University of MinnesotaCompletedMucopolysaccharidosis | Hurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Krabbe Disease | Adrenoleukodystrophy (ALD) | Hunter Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Metachromatic Leukodystrophy (MLD) | Maroteaux-Lamy SyndromeUnited States
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St. Jude Children's Research HospitalRecruitingMucopolysaccharidosis I | Severe Combined Immunodeficiency | Neuroblastoma | Neoplasm | Malignancy, HematologicUnited 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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University of California, San FranciscoRecruiting
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REGENXBIO Inc.Enrolling by invitation
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Greenwood Genetic CenterShriners Hospitals for Children; BioMarin PharmaceuticalCompletedMPS IVA | Maroteaux Lamy Syndrome | MPS VI | Morquio Syndrome AUnited States
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TakedaTakeda Development Center Americas, Inc.RecruitingHunter Syndrome | Mucopolysaccharidosis (MPS)United States