The authors studied 24 children with hypochondroplasia. Half were female and 22 out of 24 had the p.Asn540Lys variant in the fibroblast growth factor receptor 3 (FGFR3) gene. The mean age was 5.86 ...

BioMarin’s Voxzogo has been shown to increase growth rates in children with hypochondroplasia, a genetic cause of very short stature, in a phase 2 trial. Hypochondroplasia is a form of skeletal ...

In skeletal dysplasia, TYRA-300 has demonstrated positive preclinical results in achondroplasia and hypochondroplasia and TYRA has received IND clearance from the US FDA to proceed with its BEACH301 ...