Progeria drugs

Lonafarnib is the only drug available anywhere in the world approved to treat the cause of progeria, an overabundance of an abnormal protein called progerin.

To answer that need, in April 2024, PRF published the first edition of The Progeria Clinical Care Handbook, for families touched by Progeria and their doctors. From basic health facts to daily care recommendations to extensive treatment guidelines, this 131-page handbook helps answer many questions for children with Progeria throughout the world.

The U.S. Food and Drug Administration has approved the first treatment for progeria, a rare and fatal pediatric disease, characterized by dramatic, rapid aging beginning in childhood. Decades of NIH research help lead to first FDA-approved treatment for progeria

These drugs may be beneficial in progeria treatment, but in vivo extensive studies should be performed before translating them into clinical trials. We recently showed that progerin is sequestered into abnormally shaped Promyelocytic-Nuclear Bodies (PML-NB), identified as novel biomarkers in progeria.

The therapeutic potential of a new drug – MG132 – to treat progeria, a rare syndrome involving premature and accelerated ageing.

Researchers at Boston Children s Hospital are continuing progeria clinical drug trials with Zokinvy and additional experimental therapies. The trial team continues to investigate additional small molecule drugs and genetic therapies to determine if they are candidates for future clinical trials for treatment of progeria.

A drug originally developed to treat cancer could help children with progeria, a rare genetic disorder, to live longer.

The farnesyltransferase inhibitor (FTI) lonafarnib (branded as Zokinvy) is the first and only known drug treatment for children with Progeria.

Progeria treatment includes the use of a drug called lonafarnib (Zokinvy ). Originally developed to treat cancer, lonafarnib has been shown to improve many aspects of progeria. The drug has increased the average survival rate of children with the disease by two-and-a-half years.