Quick Facts
FONT-SIZE Plus   Neg
Share SHARE
mail  E-MAIL

Breakthrough Discovery: First-Ever Treatment For Progeria Proves Promising

RELATED NEWS
Trade MRK now with 
9/25/2012 12:33 AM ET

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

Editors Pick
Chipmaker Advanced Micro Devices Inc. said Thursday after the markets closed that its first quarter loss narrowed from last year, helped by strong revenue growth in its graphics and visual solutions business. The company's quarterly earnings per share, excluding items, also came in above analysts' expectations as did its quarterly revenue. After trending higher over the past few sessions, stocks showed a lack of direction throughout the trading day on Thursday. The major averages spent the session bouncing back and forth across the unchanged line before ending the day mixed. While the major averages closed on opposite sides of the unchanged line, they all showed only modest moves. U.S. Secretary Of State John Kerry said Thursday that he and his counterparts from Russia, Ukraine and the European Union have reached an agreement on a preliminary plan for ending the ongoing crisis in Ukraine. Kerry was speaking at the end of the Ukraine contact group's first meeting, comprising the foreign ministers of Russia, Ukraine, the EU and the U.S., in the Swiss city of Geneva.
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.