The Priority Review

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Gaucher's Disease Type III

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  1. Clinical trials

    Sanofi registers Phase 3 trial of venglustat vs Cerezyme in pediatric Gaucher disease type 3

    The study, titled LEAP2KIDS, plans to enroll 18 pediatric participants aged 2 to 11 years with Gaucher Disease Type III who have reached therapeutic goals with enzyme replacement therapy. It is a randomized, open-label, active-comparator trial with a 52-week primary analysis period and a 52-week extended treatment period.