Expanding horizons of achondroplasia treatment: current options and future developments.

Abstract excerpt

Activating mutations in the FGFR3 receptor tyrosine kinase lead to most prevalent form of genetic dwarfism in humans, the achondroplasia. Many features of the complex function of FGFR3 in growing skeleton were characterized, which facilitated identification of therapy targets, and drove progress toward treatment. In August 2021, the vosoritide was approved for treatment of achondroplasia, which is based on a...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Achondroplasia
• Share to Mutation
• Share to Receptor, Fibroblast Growth Factor, Type 3