Mucopolysaccharidosis I - 56 Studies Found

Completed : A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
: MPS IV A
: 2009-04-10
: Drug: BMN 110 Subjects will receive a weekly 4- to 5-hour
Enrolling by invitation : BMN 110 Phase 3B in Australian Patients
: Mucopolysaccharidosis IVA (Morquio A Syndrome)
: 2013-09-10
: Drug: BMN 110 All pateints receive treatment with BMN 110
Terminated : Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
:
  • Mucopolysaccharidosis IVA
  • Morquio A Syndrome
  • MPS IVA

    • : 2012-09-17
    : Drug: BMN 110 Drug will be delivered through a 4 hour (approximate) IV infusion at a dosage amount of 2.
Terminated : Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
:
  • Mucopolysaccharidosis IVA
  • Morquio A Syndrome
  • MPS IVA

    • : 2012-05-24
    :
    • Drug: BMN 110 Weekly IV infusi
Completed : Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
:
  • Mucopolysaccharidosis IVA
  • Morquio A Syndrome
  • MPS IVA

    • : 2011-12-22
    : Drug: BMN 110 Patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg/wk o
RECRUITING : ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
: Mucopolysaccharidosis IH/S
: 2024-11-22
: Autologous plasmablasts (B cells) engineered to express ?-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system.
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