Savarirayan R, Tofts L, Irving M, et al. - Researchers conducted this randomized, double-blind, phase 3, placebo-controlled, multicentre trial to compare once-daily subcutaneous administration of vosoritide with placebo in children with achondroplasia. The trial was performed in hospitals at 24 sites in seven countries ie, Australia, Germany, Japan, Spain, Turkey, the USA, and the UK. Participants in the study received either vosoritide 15·0 μg/kg or placebo, as assigned, for the duration of the 52-week treatment period administered via regular subcutaneous injections in their homes by qualified caregivers. All participants were selected from December 12, 2016, to November 7, 2018, with 60 assigned to receive vosoritide and 61 to receive a placebo. One hundred twenty-one patients were randomly selected out of 124 patients screened for eligibility, and 119 patients completed the 52-week trial. Findings suggested that vosoritide is an effective treatment in children with achondroplasia that can increase growth. It is not clear if the final height of adults will be raised, or what the long-term therapy impacts may be. None of the significant adverse effects is deemed treatment-related and there were no fatalities.