hrp0098p1-18 | Bone, Growth Plate and Mineral Metabolism 1 | ESPE2024

Vosoritide therapy in 16 young children with achondroplasia: a retrospective study

Adachi Natsuho , Sato Ayami , Chiba Yumiko , Tanaka Hiroyuki

Background: Vosoritide is a biological analogue of C-type natriuretic peptide, which promotes endochondral ossification by suppressing FGFR3 downstream signaling in achondroplasia (ACH). It is the first drug targeting the pathophysiology of ACH and was launched in Japan in August 2022, for the first time in the world without a minimum age limit. There have been no reports on the efficacy and safety of vosoritide other than clinical trials.<p class="abstext...