logo
Share SHARE
FONT-SIZE Plus   Neg

Breakthrough Discovery: First-Ever Treatment For Progeria Proves Promising

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) , 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.

by RTT Staff Writer

For comments and feedback: editorial@rttnews.com

Business News

Quick Facts

Editors Pick
The next iPhone to come out in 2017 may have a much clearer screen. As per reports, the iPhone 7 will use AMOLED technology in their new model, which will also sport a bigger 5.8-inch screen. It was also reported that the suppliers are already gearing up for a huge jump in orders. Best Buy Co., Inc. (BBY) reported first-quarter non-GAAP earnings per share from continuing operations of $0.44, an increase of 19% from $0.37, a year ago. On average, 21 analysts polled by Thomson Reuters expected the company to report profit per share of $0.35 for the quarter. Analysts' estimates typically... Automotive parts retailer AutoZone, Inc. on Tuesday reported a 6 percent increase in profit for the third quarter from last year, reflecting growth in sales and higher inventory on new store openings. However, both revenue and earnings per share for the quarter missed analysts' expectations.
comments powered by Disqus
Follow RTT