P216: Effectiveness of vosoritide in children with achondroplasia starting treatment aged <2 years: Interim results from a Japanese real-world study

Genetics in Medicine Open 2026 Open access

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Authors: Shirou Matsumoto, Hiroyuki Tanaka, Taichi Kitaoka, Veronika Horváthová, Jeanne M. Pimenta, Sheila Reiss Reddy, Hirofumi Tokuoka, Karen Gu, Toshimi Michigami
DOI: 10.1016/j.gimo.2026.103710
Cited by: 0

Abstract

Achondroplasia is a rare skeletal dysplasia that results in impaired endochondral bone growth from a gain-of-function variant in Fibroblast Growth Factor Receptor 3 (FGFR3). Children with achondroplasia experience multisystem complications that cause impaired function and pain that often need special intervention.

Fields of Study & Subjects

Connective tissue disorders research Growth Hormone and Insulin-like Growth Factors Craniofacial Disorders and Treatments

Publication details

Journal / source
Genetics in Medicine Open
Volume
4
Pages
103710–103710
Publication date
1 January 2026
Language
EN