A Phase 3 clinical study of UX003 (rhGUS) for the treatment of MPS VII (Sly Syndrome) shows promising results as the treatment seemed to be effective.
Reference Harmatz P, Whitley C, Wang R, et al. A novel, randomized, placebo-controlled, blind-start, single-crossover phase 3 study to assess the efficacy and safety of UX003 (rhGUS) enzyme replacement therapy in patients with MPS VII. Presented at the 13th Annual WORLDSymposium; February 13-17, 2016; San Diego, CA. http://dx.doi.org/10.1016/j.ymgme.2016.11.144