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Phase 3 randomized blind-start crossover study of UX003 (rhGUS) in MPS 7
2014-005638-71Phase 3
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Dec 2014
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 3, randomised trial in mucopolysaccharidosis type VII comparing Immediate UX003 arm with Placebo blind-start then UX003 arm (2 arms).
02
Drugs
vestronidase alfa
03
Conditions
mucopolysaccharidosis type VII
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