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A new FDA approved drug offers hope to children with progeria

Zokinvy blocks production of a defective protein to lengthen the shortened life span


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 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.

Mentioned in this article:

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Eiger BioPharmaceuticals

Biopharmaceutical company focused on the development and commercialisation of drugs for rare diseases

JAMA Network

Journal of the American Medical Association.

Monica Kleinman

Associate Professor of Anaesthesia, Harvard Medical School

Topics mentioned on this page:
Ageing Research