Overview

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

Status:
Completed

Trial end date:
2019-01-14

Target enrollment:

Participant gender:

Summary

The primary objective of the study is to evaluate the long-term safety of UX003 in subjects with MPS 7.

Phase:

Phase 3

Details

Lead Sponsor:

Ultragenyx Pharmaceutical Inc