The image is from Sundry Photography.
The FDA approved the first drug to treat a form of dwarfism called achondroplasia.
The Genetic and Rare Diseases Information Center states that achondroplasia is caused by a deficiency in the FGFR3 gene. Studies suggest that these genes push the gene into overdrive, which leads to the development of short and abnormal-shaped bones, and causes people with achondroplasia to be of short stature. In addition to dwarfism, the mutations can cause health problems, such as sleep apnea, ear infections, and a build-up of fluid in the brain, that require surgery to correct.
A treatment for achondroplasia has been developed by a U.S.-based company for years. According to the FDA, that drug has been approved for use in children ages 5 and up.
Genetics by the numbers is a story.
According to clinical trials, the drug, called Voxzogo, is safe and effective at restoring bone growth in children with achondroplasia and thus boosting their heights, but it's unclear whether the drug also counteracts the various health issues that can arise from the condition.
vosoritide works by binding to a receptor that is found in bone and cartilage tissues. According to a statement from BioMarin Pharmaceutical, the drug sets off a chain reaction that stimulates bone growth and ignores the effects of the FGFR3 mutations.
The most recent trial included 121 people who were between the ages of 5 and 18 and had their growth plates still open. The trial organizers assessed the children's height growth over the course of a year, after the participants were randomly assigned to receive either a daily injection or a placebo shot.
Injection site reactions, such as redness, vomiting, and decreased blood pressure, are the most common side effects of the treatment. By the end of the year, participants who received the drug grew an average of 0.6 inches taller than those who did not. According to BioMarin Pharmaceutical, the data shows that children with achondroplasia could reach the same heights as children without the condition.
According to BioMarin Pharmaceutical, the US should have Voxzogo in December. The treatment costs $320,000 a year.
The dwarfism community has historically drawn mixed reactions to Voxzogo. The drug seems to be aimed at "correcting" short stature, which they argue isn't a problem that requires fixing. Other little people hope that the treatment will help improve people's quality of life by warding off the health problems of achondroplasia.
Read more about the reception and approval of Voxzogo at the news website.
Live Science published the original article.
