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Treatment for Rapid Aging

The US Food and Drug Administration has approved a treatment for a genetic disorder that causes rapid aging among children, NPR reports.

The agency approved Eiger BioPharmaceuticals' Zokinvy (lonafarnib) to treat Hutchinson-Gilford progeria syndrome and certain other progeroid laminopathies. These conditions, the agency notes, lead to premature aging and death, typically due to heart failure, heart attack, or stroke, by the age of 15.

According to NPR, Sam Berns, a progeria patient who died in 2014, spurred the collection of evidence indicating Zokinvy helped progeria patients live longer. It notes that nearly every child in the US with progeria was being given Zokinvy as part of a clinical trial and that a foundation started on Berns's behalf undertook a worldwide search for data on patients who had not been given the drug to serve as a control group. 

The FDA notes that patients with Hutchinson-Gilford progeria syndrome treated with Zokinvy lived an average three months longer in the first three years of treatment and, by the maximum follow up of 11 years, lived an average 2.5 years longer.