Role of vosoritide drug on children's with achondroplasia

Abstract

Achondroplasia is a genetic condition with an autosomal dominant inheritance pattern. It is caused by detrimental heterozygous FGFR3 gene mutations. Gain-of-function mutations in the FGFR3 gene impair chondrocyte differentiation and proliferation, which has a negative impact on the formation of new bone. Vosoritide, also known as voxzogo, is the first pharmacological intervention to receive approval for the treatment of achondroplasia. This therapeutic agent has been sanctioned for administration via at-home injections, which are to be performed under the supervision of a proficient nurse.