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

IMPE Abstracts

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impe0096fc4.1 | Growth and Syndromes | IMPE2023

Persistent and stable growth promoting effects of vosoritide in children with achondroplasia for up to 3.5 years: results from an ongoing Phase 3 extension study

E. Polgreen Lynda , Savarirayan Ravi , Tofts Louise , Irving Melita , Wilcox William , A. Bacino Carlos , Hoover-Fong Julie , Ullot Font Ullot Font Rosendo , Harmatz Paul , Rutsch Frank , B. Bober Michael , Ginebreda Ignacio , Mohnike Klaus , Charrow Joel , Hoernschemeyer Daniel , Ozono Keiichi , Alanay Yasemin , Arundel Paul , Kagami Shoji , Yasui Natsuo , White Klane , M. Saal Howard , Leiva-Gea Antonio , Luna-González Felipe , Mochizuki Hiroshi , Basel Donald , M. Porco Dania , Jayaram Kala , Fisheleva Elena , Lawrinson Sue , Day Jonathan

Objectives: Vosoritide is a potent stimulator of endochondral bone growth and is in development for the treatment of achondroplasia, the most common form of disproportionate short stature. We previously reported on a 52-week, phase 3, pivotal study that demonstrated a significant improvement in annualized growth velocity (AGV) when vosoritide was compared to placebo in children with achondroplasia aged 5-18 years (Savarirayan et al, Lancet, 2020). Thi...
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