Key points from article :
FDA has approved a treatment that could give children with progeria more time to live.
The drug - Zokinvy - is the first and only approved treatment for progeria.
Children with progeria have higher amounts of a defective protein progerin causing the cells to prematurely age.
Zokinvy blocks some but not all of that progerin production.
Most children with the disorder die before they reach age 15.
The drug increased life span by 3 months during the first 3 years of treatment in a clinical trial of 62 children.
Continuing the drug for up to 11 years, life spans were lengthened by 2.5 years.
"We’ve extended the life span...by slowing the pace of the disease,” but the drug doesn’t give kids a normal length of life. - Dr. Monica Kleinman, lead researcher.
The focus is now to test additional drugs or therapeutics in combination with Zokinvy.
Research by Eiger BioPharmaceuticals published in JAMA Network.
