hrp0097rfc4.5 | Growth and syndromes (to include Turner syndrome) | ESPE2023
Cormier-Daire Valérie
, Edouard Thomas
, Isidor Bertrand
, Cohen Shelda
, Mukherjee Swati
, Pimenta Jeanne
, Lhaneche Leila
, Rossi Massimiliano
, Schaefer Elise
, Goodman Erin
, Sigaudy Sabine
, Baujat Geneviève
Introduction: Achondroplasia is the most common skeletal dysplasia, in which the main clinical feature is short stature. Vosoritide, the first specific treatment for achondroplasia; administered as a daily subcutaneous injection, was approved by the European Medicines Agency in August 2021 for patients aged ≥2 years until closure of epiphyses. French Health Authorities granted early access to vosoritide treatment in France on 24 June 2021, which continued u...