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hrp0097fc4.6 | Growth and syndromes (to include Turner syndrome) | ESPE2023

Persistence of Growth Promoting Effects in Infants and Toddlers with Achondroplasia: Results in Children Aged Over 2 Years Old from a Phase II Extension Study with Vosoritide

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

Introduction: Vosoritide increases growth in children with achondroplasia aged 5–18 years (Savarirayan et al, Genet Med, 2021). We recently reported results from a phase 2, randomized, double-blind, placebo-controlled study (111-206) in young children with achondroplasia aged 3 months–5 years Here we present results from an open-label extension study (111-208) spanning up to 4 years of treatment in the children aged 2-5 years at study star...

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Persistence of Growth Promoting Effects in Infants and Toddlers with Achondroplasia: Results in Children Aged Over 2 Years Old from a Phase II Extension Study with Vosoritide

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