Hutchinson-Gilford progeria syndrome, otherwise known as progeria, is a rare disorder that causes accelerated aging in children and affects 350 to 400 children around the globe. This genetic disorder is caused by a mutation that results in high levels of a defective form of the protein lamin A, which is responsible for holding a cell’s nucleus together. Although this faulty protein, called progerin, is produced more as we age, excessive amounts of progerin contributes to the symptom of joint stiffness in kids with progeria by stiffening connective tissue and blood vessels. Although there is no cure for progeria yet, the FDA has approved the first ever drug treatment for progeria, and it is called Zokinvy. When children with progeria were treated with Zokinvy in clinical trials, researchers found that the drug lengthened the children's lifespans by an average of 3 months when it was used as a treatment for 3 years and lengthened them by an average of 2.5 years when the treatment was used for up to 11 years. Zokinvy works to inhibit the production of progerin and thus, decelerates the rapidness of aging that would normally be observed. This newly approved drug shows very promising results and in the next step, researchers are hoping to discover a more effective treatment by combining Zokinvy with other therapeutics.
Wilke, Carolyn. “The FDA Has Approved the First Drug to Treat the Rapid-Aging Disease Progeria.” Science News, 25 Nov. 2020, www.sciencenews.org/article/fda-approved-first-drug-treat-rapid-aging-disease-progeria.