Savarirayan R, et al. - Through a multinational, phase 2, dose-finding study and extension study with 35 children, researchers analyzed the safety and side-effect profile of vosoritide in children aged from 5 to 14 years with achondroplasia, a genetic disorder that impedes endochondral ossification, causing disproportionate short stature and substantial medical complications. Adverse events were noted in all 35 patients, with serious adverse events noted in 4 of 35. Discontinuation of therapy was needed in 6 patients. A mild side-effect profile of once-daily subcutaneous administration of vosoritide was observed in children with achondroplasia. Further, a sustained increase in the annualized growth velocity for up to 42 months via the treatment was also noted.