Quick Facts
FONT-SIZE Plus   Neg
Share SHARE

Breakthrough Discovery: First-Ever Treatment For Progeria Proves Promising

RELATED NEWS
Trade MRK now with 

More than nine years have elapsed since scientists discovered the cause of Progeria - the deadly genetic condition of premature aging in children. Although as yet there is no cure, a drug originally developed for cancer has demonstrated efficacy in the first-ever clinical drug trial for children with Progeria.

Progeria, which is derived from the Greek word for old age, is an extremely rare disease and is estimated to affect approximately 1 in 4 - 8 million newborns. Children with progeria may suffer heart attacks and strokes as early as age 5 years, and their average life expectancy is just 13 years.

In the two-and-a-half year drug trial, twenty-eight children from sixteen countries were enrolled and they were treated with farnesyltransferase inhibitor lonafarnib, supplied by Merck & Co. (MRK: Quote) , given orally, twice-a-day over the course of the study.

According to the trial results, there were significant improvements in weight gain, bone structure and, most importantly, the cardiovascular system of the children.

Progeria is caused by a mutation in the gene called LMNA that produces a progerin protein, damaging the cell function. In children with progeria, a molecule called a "farnesyl group" gets attached to the progerin protein. The farnesyltransferase inhibitor drug is known to work by blocking the attachment of the farnesyl group onto progerin.

The study results were published September 24 in Proceedings of the National Academy of Sciences.

Register
To receive FREE breaking news email alerts for Merck & Co Inc. and others in your portfolio

by RTT Staff Writer

For comments and feedback: editorial@rttnews.com

Business News

Quick Facts

Editors Pick
Colorado based doctor Dilip Joseph, who was kidnapped by the Taliban discusses his ordeal in detail in a book published by Amazon on Tuesday. Extending the rebound seen over the course of the previous session, stocks moved sharply higher during trading on Friday. The gains on the day partly offset the recent sell-off on Wall Street, although the major averages remain well off their mid-September highs. While the Ebola outbreak in the U.S. has thus far been limited to just three people, the results of a new CBS News poll suggest that news of the disease has contributed to a sharp drop in public confidence in the Centers for Disease Control and Prevention.
comments powered by Disqus
FREE Newsletters, Analysis & Alerts

 

Stay informed with our FREE daily Newsletters and real-time breaking News Alerts. Sign up to receive the latest information on business news, health, technology, biotech, market analysis, currency trading and more.