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MPS II
Clinical trial pipeline · Data from ClinicalTrials.gov
See which MPS II trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which MPS II trials you may qualify forThe main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the dise…
For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and…
The goal of this observational study is to characterize the epidemiology and natural history of MPS diseases by building a retrospective and prospective collect…
The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understandin…
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen pai…
This is a multiregional open-label extension (OLE) to assess the safety, tolerability, and efficacy of long-term treatment with tividenofusp alfa (DNL310), an i…
A Global Phase III multicenter, randomized, assessor-blinded, active-controlled designed to evaluate safety and efficacy of study drug for the treatment of the…