P222: From early access to routine care: Prospective real-world follow-up of vosoritide in achondroplasia in France

Genetics in Medicine Open 2026 Open access

Read full text
Authors: Geneviève Baujat, Elise Schaefer, Massimiliano Rossi, Marc-Antoine Hamandjian, Laura Pauget, Victor Morel, Marjolaine Willems, Thomas Édouard, Valérie Cormier‐Daire
DOI: 10.1016/j.gimo.2026.103716
Cited by: 0

Abstract

Achondroplasia is the most common form of skeletal dysplasia, characterized by disproportionate short stature caused by a gain-of-function pathogenic variant in the fibroblast growth factor receptor 3 (FGFR3) gene. Vosoritide, a recombinant C-type natriuretic peptide, is the first targeted therapy approved for children with achondroplasia (EMA, 2021), showing a favorable safety profile and improvements in bone growth and quality of life.

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
103716–103716
Publication date
1 January 2026
Language
EN