ln a tral in children and adoles-cents with achondroplasia, aged 5 to 15 years, an increase in growth rate of about 1.5 cm per year was observed with vosoritide compared to placebo. The follow-up period is too short to determine its potential effect on adult height, or on other clinical manifestations of the disease and its complications. As of late 2022, the data in children aged 2 to 5 years are insufficient. Vosoritide (a recombinant form of C-type natriuretic peptide) mainly carries a risk of injectionsite reactions and reductions in blood pressure. Given the safety signals of bone malformation and osteonecrosis seen in animals, patients should be closely monitored for the occurrence of long-term adverse effects. As of late 2022, the observed effect of vosoritide on growth justifies continuing its clinical evaluation over a longer period.
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