Preidt, Robert. (June 21, 2019) New Drug Combats Leading Cause of Dwarfism. U.S. News & World Report. Available at https://www.usnews.com/news/health-news/articles/2019-06-21/new-drug-combats-leading-cause-of-dwarfism (Accessed June 23, 2019).
The intervention that the article focused on was using a drug called vosoritide to regulate the bone development in children with the genetic bone disorder achondroplasia. Achondroplasia is the most common form of dwarfism. In the United States, France, United Kingdom and Australia a four-year phase 2 trial was conducted. 35 children aged 5-14 were separated into four groups and given daily doses of vosoritide in increasing quantities. This caused the children to increase in height an average of 2.4 inches a year. It increased the children’s growth rate on average by 50%. The side effects of the drug were generally minor. I am convinced of the effectiveness of this intervention because of the increased growth rate of the children with only minor side effects. Achondroplasia affects approximately 1 in 25,000 infants and is caused by a FGFR3 gene mutation which adversely affects bone growth. An overactive signal that stops development is what causes achondroplasia. This intervention is effective because vosoritide helps to stop that overactivity thus helping to solve the problem. The intervention is likely to catch on because from a policy perspective it is addressing the needs of these children by improving their future health on a yearly basis. The key decision makers are likely to agree to recommend and fund this intervention because it increased the growth rates of children with achondroplasia per year to nearly the same growth rates per year of children of average height. Many children with achondroplasia have other complications such as spinal chord compression, spinal curvature and bowed legs. These complications require corrective surgeries in approximately half of the children. This intervention may improve the children’s health outcomes, functioning and access to their environment which would increase the likelihood that it would be funded by key decision makers.
I think it’s cool that there is a drug that could potentially get rid of drafism for good, but I have to wonder if it does pass, will the drug be avaible for people who want to increase their height for non-medical reasons. To be more precise, would it be used for people who are unsatisfied with their height and want to change that.
Wow, that’s amazing. Not only will it help children, but it will also help their parents who may not be able to afford the corrective surgeries for the complications dwarfism causes. I wonder how policymakers will regulate the use and perscription of the drug