hrp0098p2-56 | Bone, Growth Plate and Mineral Metabolism | ESPE2024
Cormier-Daire Valerie
, Edouard Thomas
, Isidor Bertrand
, Pimenta Jeanne
, Mukherjee Swati
, Marcos Valeria
, Dee Anne
, Rossi Massimiliano
, Schaefer Elise
, Sigaudy Sabine
, Baujat Geneviève
Introduction: Achondroplasia is the most common skeletal dysplasia, caused by a pathogenic FGFR3 variant, leading to impaired endochondral bone growth and multiple medical complications. Vosoritide, a modified recombinant human C-type natriuretic peptide (rhCNP), was first approved by the European Medicines Agency in August 2021 and is now approved for treating genetically-confirmed achondroplasia in patients aged ≥4 months until closure of epiphys...