According to a story from EurekAlert, a recent study published in the journal Pediatric Research reveals new protein biomarkers that researchers can use to evaluate how patients with progeria have reacted to treatment. There are no treatments that have proven to be effective in halting the effects of the condition.
Progeria is an incredibly rare genetic disorder in which the sufferer appears to experience the effects of aging at an unusually rapid rate and at a very young age. It mostly occurs as a new mutation as its carriers typically die before they can pass on the mutation to anyone else; most people with progeria survive into their teens or twenties. A child with progeria begins to develop early symptoms within a few months, and by their first year they have developed a distinctive appearance. Growth is limited, and all hair is lost. Later, problems with the heart, kidneys, and eyesight begin to develop. Treatment mostly focuses on mitigating the complications that the sufferer experiences as they develop. To learn more about progeria, click here.
The drug lonafarnib has shown some degree of usefulness in progeria patients. It allows progeria patients to maintain and gain weight and offers relief from headaches. It also reduces the risk of heart attack. However, researchers had no way of quantifying the positive effects of treatment or determining the nature of differences in patient response. Samples from 24 patients were taken from before and after treatment with lonafarib. When compared to a control group of healthy children, there were differences in levels of about 40 percent of the tested proteins.
