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Mucopolysaccharidosis I - 57 Studies Found

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

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

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

    • Date: 2011-12-22
    Interventions: Drug: BMN 110 Patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg/wk o
Completed Study Name: ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
Condition: Mucopolysaccharidosis IH/S
Date: 2025-08-05
Interventions: Autologous plasmablasts (B cells) engineered to express ?-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system.
COMPLETED Study Name: A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)
Condition: Mucopolysaccharidosis III-A
Date: 2025-08-05
Interventions: No intervention
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