ESPE Abstracts

ESPE Abstracts

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Real World Effectiveness of Vosoritide in 165 Children with Achondroplasia – Data from a Multicenter European Registry

Wechsung Katja , Al-Halak Maesa , Avdjieva-Tzavella Daniela , Bechtold-Dalla Pozza Susanne , Beger Christoph , Gausche Ruth , Grasemann Corinna , Högler Wolfgang , Hösl Matthias , Hoyer-Kuhn Heike , Huebner Angela , Ingwers Anja , Janner Marco , Keller Alexandra , Kunkel Philip , Nader Sean , Mohnike Klaus , Muschol Nicole , Palm Katja , Pötzsch Simone , Rutsch Frank , Schnabel Dirk , Soucek Ondrej , Streata Ioana , Vogt Björn , MK Völkl Thomas , Wölfle Joachim , Zeman Jiri , Pfäffle Roland

Introduction: Vosoritide is licensed by EMA for the treatment of children with achondroplasia above 4 months of age in 2023 (initial approval for children above 2 years in 2021). The CNP analogue modulates enchondral bone growth and improves growth velocity. Response to therapy varies between individuals. Nineteen European centers enter auxological and diagnosis-specific data into the achondroplasia module of the CrescNet® registry to monitor treatment. We...
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ESPE Abstracts

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