Long-term vosoritide treatment sustains bone growth and may enhance wellbeing in children with achondroplasia: a plain language summary

Plain Language Summary: Background Achondroplasia is a skeletal dysplasia, which is a condition that affects bones and joints. It causes reduced height because bones do not grow in the typical way and lead to bowed legs and back problems. Reduced bone growth in achondroplasia can also cause other medical issues such as interrupted breathing when sleeping, hearing difficulties, pain and obesity. Vosoritide is a treatment designed to increase bone growth of children with achondroplasia. In a 2020 study, children treated with vosoritide for 1 year had an increased rate of bone growth compared with those who did not receive vosoritide. The side effects were mild, with the most common being a rash or redness where the injection was given. Those who completed this study could choose to go into an extended phase of the study to look at results and safety of vosoritide treatment over a longer period of time. What is this summary about? This summary reports the results of vosoritide treatment for at least 3 years (and up to 6 years) in 2 parts: Part A looks at the effects of treatment on the growth of children with achondroplasia till puberty or when their bones stopped growing, and Part B looks at how growth affected how they felt, functioned and were able to enjoy life (wellbeing). The study showed that vosoritide treatment, for at least 3 years, helped improve bone growth in children with achondroplasia. It also improved how they felt physically and socially. Side effects of vosoritide were mostly mild and got better on their own. The most common side effect was a redness at the injection site. What do the results mean? Long-term treatment with vosoritide can increase bone growth in children with achondroplasia and may have a positive impact on their everyday wellbeing. How to say (download PDF and double click sound icon to play sound)… Achondroplasia: Ay-kon-droh-play-zee-ah Dysplasia: Diss-play-zee-ah Vosoritide: Voh-sor-i-tied This is an abstract of the Plain Language Summary of Publication article. View the full Plain Language Summary PDF of this article to read the full-text Link to original article here and here Trial registration:ClinicalTrials.gov identifier: NCT03424018.