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
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Karen Jacobs | C. Whitley | P. Harmatz | Raymond Y. Wang | E. Schwartz | C. Haller | E. Kakkis | M. Bauer | Wenjie Song