hrp0098p3-164 | Growth and Syndromes | ESPE2024

Design and objectives of Study 111-902: a multicenter, prospective and retrospective observational study of children with hypochondroplasia

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...