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P141: Persistent Growth-Promoting Effects of Vosoritide in Children with Achondroplasia is Accompanied by Improvement in Physical Aspects of Quality of Life*

Ravi Savarirayan,Louise Tofts,Melita Irving,William Wilcox,Carlos Bacino,Julie Hoover-Fong,Rosendo Ullot Font,Paul Harmatz,Frank Rutsch,Ricki Carroll,Lynda Polgreen,Ignacio Ginebreda,Klaus Mohnike,Joel Charrow,Carlos Prada,Daniel Hoernschemeyer,Keiichi Ozono,Takuo Kubota,Yasemin Alanay,Paul Arundel

Genetics in Medicine Open（2024）

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Abstract
Vosoritide, a potent stimulator of endochondral bone growth, is approved for use in children with achondroplasia (ACH) until closure of epiphyses. A 52-week, phase 3, pivotal trial (111-301) demonstrated a significant improvement in annualized growth velocity (AGV) with vosoritide compared to placebo in children with ACH aged 5-18 years. We report data from Quality of Life in Short Stature Youth (QoLISSY) questionnaires collected for treated children in study 111-301 and its ongoing open-label extension study 111-302.
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