Phase 3 Study of ERT for MPS VII (Sly disease)

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

 

Author: Ashok Vellodi

I have had a lifelong interest in lysosomal storage disorders. Having recently retired I now have time to pursue my passion for teaching and training of young doctors and scientists, and hopefully instil in them the passion for their patients that is so integral to working in this field.

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