hrp0098p3-164 | Growth and Syndromes | ESPE2024
Dauber Andrew
, Irving Melita
, Savarirayan Ravi
, Ghani Sajda
, Sabir Ian
, Bassi Roberto
Introduction: Hypochondroplasia is a rare genetic growth-related condition primarily caused by FGFR3 variants that lead to decreased endochondral bone growth, disproportionate short stature and other medical complications. It shares pathogenetic and phenotypic similarities to achondroplasia. Outside of Japan, there are no approved pharmacological treatments for hypochondroplasia. Vosoritide, a C-type natriuretic peptide analog, is an approved first-in...