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hrp0095p1-223 | Bone, Growth Plate and Mineral Metabolism | ESPE2022

Real-world data in children with achondroplasia after licensing of Vosoritide

Palm Katja , Bechthold-Dalla Pozza Susanne , Gausche Ruth , Högler Wolfgang , Hoyer- Kuhn Heike , Hübner Angela , Keller Alexandra , Mirante Alice , Mohnike Klaus , Muschol Nicole , Nader Sean , Pfäffle Roland , Quitter Friederike , Rohrer Tilmann , Rutsch Frank , Schnabel Dirk , Semler Oliver , Silva Isabel , B. Sousa Sérgio , M.K. Voelkl Thomas , Wechsung Katja , Weigel Johannes , Woelffle Joachim , Lausch Ekkehart

Background: Achondroplasia (ACH), caused by a pathogenic variant in the fibroblast growth factor receptor 3 gene (FGFR3), is characterized by severe growth failure and may be associated with multisystemic complications. The clinical phenotype is variable and relates to deformity of rhizomelic shortened legs, and myelon compression at cranial base and spine. Recent guidelines are published for diagnostic workflow, neurosurgical, orthopaedic and otorhinolaryngol...

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hrp0098t20 | Top 20 Posters | ESPE2024

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

Real-world data in children with achondroplasia after licensing of Vosoritide

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

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