Scientific Programme & Abstracts from the International Meeting in Pediatric Endocrinology (IMPE)

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impe0096p92 | Growth and Syndromes | IMPE2023

A Randomized Controlled Trial of Vosoritide in in Infants and Toddlers with Achondroplasia

E. Polgreen Lynda , Savarirayan Ravi , W. Wilcox William , Harmatz Paul , Phillips John , III , Tofts Louise , Ozono Keiichi , Arundel Paul , Irving Melita , A. Bacino Carlos , Basel Donald , B. Bober Michael , Charrow Joel , Mochizuki Hiroshi , Kotani Yumiko , M. Saal Howard , Jeha George , Han Lynn , Fisheleva Elena , Huntsman-Labed Alice , Day Jonathan

Background: Vosoritide increases annualized growth velocity (AGV) in children with achondroplasia aged 5 to 18 years. This global, phase 2, randomized, double-blind, placebo-controlled study evaluated the safety and efficacy of vosoritide on growth in children with achondroplasia aged 3 months to <5 years.Methods: This study compared once-daily subcutaneous administration of vosoritide, at doses of 15 or 30 mg/kg of b...
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