Dosing:
This is an open label study, in which participants will be sequentially assigned to receive either 0.75 mg/kg or 1.5 mg/kg of PPS via subcutaneous injection. PPS will be administered weekly for the first 12 weeks and then every second week until the end of the study.
The study will run for 49 weeks from baseline. Following the screening visit, the participants will be required to attend the hospital for assessments out to week 49.
Reference: ANZCTR Site
Ann: Preliminary Results from Phase 2 MPS I Study, page-15
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