The aim of the study is to assess the safety of the investigational product pentosan polysulfate (PPS) in patients with MPSI, and to determine if PPS can successfully alleviate pain and functional symptoms in MPS I patients who have received ERT and/or haemopoietic stem cell transplantation (HSCT).
https://www.anzctr.org.au/Trial/Registration/TrialReview.aspx?id=379536&isReview=true