Example: Heart Attack

Clinical Trials on Mucopolysaccharidosis VII

Total 13 results





    • NCT04532047
      Not yet recruiting
      In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases
      Conditions: MPS I; MPS II; MPS IVA; MPS VI; Mps VII; Gaucher Disease, Type 2; Gaucher Disease, Type 3; Pompe Disease Infantile-Onset; Wolman Disease
    • NCT03775174
      Available
      Expanded Access to Mepsevii
      Conditions: MPS VII; Mucopolysaccharidosis VII; Sly Syndrome
    • NCT03604835
      Recruiting
      Mucopolysaccharidosis VII Disease Monitoring Program
      Conditions: Mucopolysaccharidosis VII; MPS VII; MPS 7; Sly Syndrome
    • NCT02432144
      Completed
      A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)
      Conditions: Sly Syndrome; MPS VII; Mucopolysaccharidosis; Mucopolysaccharidosis VII
    • NCT02418455
      Completed
      Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age
      Conditions: Sly Syndrome; MPS VII; Mucopolysaccharidosis; Mucopolysaccharidosis VII
    • NCT02298699
      Active, not recruiting
      Biomarker for Sly Disease (MPS VII) (BioSly)
      Conditions: Developmental Delay; Skeletal Abnormalities; Hepatomegaly; Splenomegaly
    • NCT02230566
      Completed
      A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
      Conditions: MPS 7; Sly Syndrome; Mucopolysaccharidosis; MPS VII
    • NCT02171104
      Recruiting
      MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
      Conditions: Mucopolysaccharidosis Disorders; Hurler Syndrome; Hunter Syndrome; Maroteaux Lamy Syndrome; Sly Syndrome; Alpha-Mannosidosis; Fucosidosis; Aspartylglucosaminuria; Glycoprotein Metabolic Disorders; Sphingolipidoses; Recessive Leukodystrophies; Globoid Cell Leukodystrophy; Metachromatic Leukodystrophy; Niemann-Pick B; Niemann-Pick C Subtype 2; Sphingomyelin Deficiency; Peroxisomal Disorders; Adrenoleukodystrophy With Cerebral Involvement; Zellweger Syndrome; Neonatal Adrenoleukodystrophy; Infantile Refsum Disease; Acyl-CoA Oxidase Deficiency; D-Bifunctional Enzyme Deficiency; Multifunctional Enzyme Deficiency; Alpha-methylacyl-CoA Racmase Deficiency; Mitochondrial Neurogastrointestingal Encephalopathy; Severe Osteopetrosis; Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation); Inherited Metabolic Disorders
    • NCT02097251
      No longer available
      An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
      Conditions: Mucopolysaccharidosis Type 7
    • NCT01870375
      Completed
      Longitudinal Studies of Brain Structure and Function in MPS Disorders
      Conditions: Mucopolysaccharidosis Type I; Mucopolysaccharidosis Type II; Mucopolysaccharidosis Type VI; Mucopolysaccharidosis Type IV; Mucopolysaccharidosis Type VII
    • NCT01856218
      Completed
      An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
      Conditions: Mucopolysaccharidosis Type 7
    • NCT01043640
      Completed
      Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
      Conditions: Mucopolysaccharidosis; Hurler Syndrome; Hunter Syndrome; Maroteaux-Lamy Syndrome; Sly Syndrome; Alpha Mannosidosis; Fucosidosis; Aspartylglucosaminuria; Adrenoleukodystrophy (ALD); Krabbe Disease; Metachromatic Leukodystrophy (MLD); Sphingolipidoses; Peroxisomal Disorders
    • NCT00668564
      Terminated
      Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
      Conditions: Hurler's Syndrome; Maroteaux-Lamy Syndrome; Sly Syndrome; Alpha Mannosidosis; Fucosidosis; Aspartylglucosaminuria; Sphingolipidoses; Krabbe Disease; Wolman's Disease; Niemann-Pick Disease Type B; Niemann-Pick Disease, Type C
      Medical Conditions
      Sponsors and Collaborators
      Drug Interventions
      Conditions
      Rare Diseases
      Drug Interventions
      Dietary Supplements
      Sponsor/Collaborators
      Locations