Therapy Against Progeria

June 7, 2018  Source: Ddu 114

Progeria or premature aging is a rare genetic disorder where the patients survive only up till 16 – 18 years. With this disease, they face age-related symptoms like baldness, the appearance of wrinkles, joint pain and cardiovascular issues.

After conducting a clinical trial study at Boston Children’s Hospital, scientists from Brown University recommended that a lonafarnib therapy could increase the survival rate of progeria patients. But the mode of action of lonafarnib is still uncertain.

The research team head Dr. Leslie Gordon said, “This study provides supporting evidence that we can begin to put the brakes on the rapid aging process for children with progeria. These results provide new promise and optimism to the progeria community.”

Dr. Francis Collins, director of the National Institutes of Health was not involved in the study. But it was his lab where the progeria causing mutation was first identified. Collins said, “My lab did some of the original research on cellular and mouse models that showed the potential benefit of this class of drugs for Progeria; it was encouraging to see those results translated into a clinical trial. Yet demonstrating the effectiveness of treatments in this small population of children with this rare fatal disease is a major challenge. Thus, I’m particularly encouraged by these latest findings.”

By Ddu
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