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 dysplasia caused by a defect in the FGFR3 gene that affects the conversion of cartilage into bone, resulting in shortened limbs and other complications including spinal problems and leg bowing.
