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Mucopolysaccharidosis VII - 10 Studies Found

Status Study
Recruiting Study Name: Biomarker for Sly Disease
Condition:
  • Mucopolysaccharidosis VII
  • Sly Disease Date: 2014-10-23
Active, not recruiting Study Name: A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7
Condition:
  • Sly Syndrome
  • MPS VII
  • Date: 2015-04-22
    Interventions: Drug: UX003 UX003 is a sterile liquid buffered saline formulation of rhGUS that contains enzyme at a con
Completed Study Name: A Phase 3 Study of UX003 rhGUS Enzyme Replacement Therapy in Patients With MPS 7
Condition:
  • MPS 7
  • Sly Syndrome
  • Date: 2014-08-22
    Interventions:
    • Drug: UX003 UX003 is a sterile
Active, not recruiting Study Name: An Open-Label Study of UX003-rhGUS Enzyme Replacement Treatment in MPS 7 Patients Less Than 5 Years of Age
Condition:
  • Sly Syndrome
  • MPS VII
  • Date: 2015-04-12
    Interventions: Drug: UX003 Other Names:
    • recombinant human beta-glucoronidase
Completed Study Name: 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 MPS 7
Condition: Mucopolysaccharidosis Type 7
Date: 2013-05-08
Interventions: Drug: UX003
No longer available Study Name: An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
Condition: Mucopolysaccharidosis Type 7
Date: 2014-03-24
Interventions: Drug: UX003 Open Label Other Names:
Recruiting Study Name: Longitudinal Studies of Brain Structure and Function in MPS Disorders
Condition:
  • Mucopolysaccharidosis Type I
  • Mucopolysaccharidosis Type II
  • Date: 2011-08-02
Terminated Study Name: Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
Condition:
  • Hurler's Syndrome
  • Maroteaux-Lamy Syndrome
  • Procedure: Stem Cell Transplantation

Recruiting Study Name: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Condition:
  • Mucopolysaccharidosis Disorders
  • Hurler Syndrome
  • Hunter Syn
    Date: 2014-06-20
    Interventions:
    • Biological: Stem Cell Transplantation

Active, not recruiting Study Name: Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Condition:
  • Mucopolysaccharidosis
  • Hurler Syndrome
  • Hunter Syndrome

    • Date: 2010-01-05
    Interventions:
    • Drug: Campath-1H Administered