Mucopolysaccharidosis I - 57 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 :
: 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 :
: 2012-05-24 : |
| Completed |
: Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome) :
: 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 |
| Completed |
: ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I : Mucopolysaccharidosis IH/S : 2025-09-08 : Autologous plasmablasts (B cells) engineered to express ?-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system. |
| COMPLETED |
: A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome) : Mucopolysaccharidosis III-A : 2025-09-08 : No intervention |