Mucopolysaccharidoses - 19 Studies Found

Completed : Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis
: Mucopolysaccharidosis
: 2010-11-08
:
  • Drug: Conditioning regimen

Completed : Natural History Studies of Mucopolysaccharidosis III
:
  • Mucopolysaccharidosis Type IIIA
  • Mucopolys
    : 2013-11-26
    :
    • Procedure: Lumbar puncture
    • Device: Magneti
Completed : A Study of JR-141 in Patients With Mucopolysaccharidosis Type II
: Mucopolysaccharidosis II
: 2017-04-10
: Drug: JR-141 IV infusion (lyophilized powder), 0.01-2.0 mg/kg/week
Completed : Study of rhASB in Patients With Mucopolysaccharidosis VI
: Mucopolysaccharidosis VI
: 2005-02-24
:
  • Drug: N-acetylgalactosamine 4-sulfatase

Terminated : Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
: Mucopolysaccharidosis Type IVA
: 2012-11-21
Completed : Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
:
  • Mucopolysaccharidosis IV A
  • Mucopolysaccha
    : 2012-10-12
    : Other: Enzyme testing Leukocyte activity measurement of Arylsulfatase B and N acetyl galactosamine 6 sul
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 MPS 7
: Mucopolysaccharidosis Type 7
: 2013-05-08
: Drug: UX003
Completed : Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)
: Mucopolysaccharidosis II
: 1999-10-18
: Genetic: lymphocyte gene therapy
Completed : Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI
:
  • Mucopolysaccharidosis Type I
  • Mucopolysacc
    : 2015-05-05
    :
    • Drug: Adalimumab Subjects will
Completed : A Treatment Study of Mucopolysaccharidosis Type IIIB
:
  • MPS III B
  • Mucopolysaccharidosis Type IIIB

: 2016-02-27
: Drug: BMN 250 Chimeric fusion of recombinant human alpha-N-acetylglucosaminidase and truncated human ins
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