Clinical Trials on Mucopolysaccharidosis I
Total 1925 results
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Ultragenyx Pharmaceutical IncCompletedMucopolysaccharidosis | Sly Syndrome | Mucopolysaccharidosis VII | MPS VIISpain, United States, Portugal
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Lundquist Institute for Biomedical Innovation at...Not yet recruitingMucopolysaccharidosis III | Sanfilippo SyndromeUnited States
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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 IncPRA Health SciencesRecruitingSly Syndrome | Mucopolysaccharidosis VII | MPS VII | MPS 7United States, Germany, Brazil, Argentina, France, Netherlands, Portugal, Spain, Turkey
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Ultragenyx Pharmaceutical IncCompletedMucopolysaccharidosis | Sly Syndrome | MPS VII | MPS 7United States
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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 IV A | Mucopolysaccharidosis IVAFrance, United Kingdom, Taiwan, United States, Argentina, Netherlands, Canada, Brazil, Germany, Italy
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JCR Pharmaceuticals Co., Ltd.Active, not recruitingMucopolysaccharidosis IIJapan
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JCR Pharmaceuticals Co., Ltd.Completed
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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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Birmingham Women's and Children's NHS Foundation...BioMarin PharmaceuticalWithdrawnMucopolysaccharidosis IV | Morquio A DiseaseUnited Kingdom
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Ultragenyx Pharmaceutical IncCompletedMucopolysaccharidosis | Sly Syndrome | Mucopolysaccharidosis VII | MPS VIIUnited States, Brazil, Mexico, Portugal
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BioMarin PharmaceuticalCompletedMucopolysaccharidosis VI | Maroteaux-Lamy SyndromeUnited States, Portugal, France
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AO GENERIUMRecruitingMetabolic Diseases | Mucopolysaccharidosis Type IIRussian Federation
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Denali Therapeutics Inc.Enrolling by invitationMucopolysaccharidosis IIUnited States, Canada, Belgium, Czechia, Netherlands
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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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ShireCompletedSanfilippo Syndrome Type BUnited States, United Kingdom, Germany
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BioMarin PharmaceuticalCompletedMucopolysaccharidosis IVA (Morquio A Syndrome)Australia
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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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AO GENERIUMCompletedA Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy VolunteersMetabolic Diseases | Mucopolysaccharidosis Type IIRussian Federation
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Nadia Ali, PhDBioMarin PharmaceuticalCompletedMucopolysaccharidosis IV | Morquio DiseaseUnited States
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ShireCompletedHunter Syndrome | Mucopolysaccharidosis (MPS)United States, Argentina, Mexico, Spain, United Kingdom
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TakedaTakeda Development Center Americas, Inc.RecruitingHunter Syndrome | Mucopolysaccharidosis (MPS)United States
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Manchester University NHS Foundation TrustOrchard TherapeuticsRecruitingMucopolysaccharidosesUnited Kingdom
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Hacettepe UniversityCompletedMucopolysaccharidosesTurkey
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Mackay Memorial HospitalBioMarin PharmaceuticalUnknown
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Assiut UniversityUnknown
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Tehran University of Medical SciencesUnknownMucopolysaccharidosisIran, Islamic Republic of
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Shrouk Shaban BakrRecruiting
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ShireTerminatedMucopolysaccharidosis (MPS)United States, Canada, United Kingdom, Turkey, Italy, Mexico, Spain
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Manchester University NHS Foundation TrustCompletedMucopolysaccharidosis Type IIUnited Kingdom
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Manchester Royal Eye HospitalBioMarin PharmaceuticalUnknownMucopolysaccharidosesUnited 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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ShireCompletedHunter Syndrome | Mucopolysaccharidosis II (MPS II)United States, France, United Kingdom, Germany, Sweden, Spain, Brazil, Canada, Italy, Romania
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St. Jude Children's Research HospitalRecruitingMucopolysaccharidosis I | Severe Combined Immunodeficiency | Neuroblastoma | Neoplasm | Malignancy, HematologicUnited States
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Hasan Kalyoncu UniversityCompleted
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Denali Therapeutics Inc.CompletedMucopolysaccharidosis IIUnited Kingdom, United States, Italy, Netherlands
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JCR Pharmaceuticals Co., Ltd.Completed
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Sangamo TherapeuticsTerminatedMucopolysaccharidosis II | MPS IIUnited States
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REGENXBIO Inc.Withdrawn
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Green Cross CorporationCompletedMucopolysaccharidosis IIKorea, Republic of
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Eunice Kennedy Shriver National Institute of Child...University of MinnesotaCompleted
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University Hospital, ToursCompletedMucopolysaccharidosesFrance
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University of California, San FranciscoRecruiting
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BioMarin PharmaceuticalCompletedMPS VIUnited States, Australia, France, Brazil, Germany, Portugal, United Kingdom
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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.Active, not recruitingMucopolysaccharidosis IIUnited States, Netherlands, Canada, United Kingdom
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JCR Pharmaceuticals Co., Ltd.Completed
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REGENXBIO Inc.Enrolling by invitation