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
Crypto Co., engaged in the digital currencies and blockchain sector, announced its plans to execute a 10-for-1 stock split, citing the significant growth in the value and demand for its shares amid the ongoing digital currency market boost. CEO Mike Poutre said, ""We are aware of the recent fluctuation in our stock, and want to see orderly market activity surrounding the trading of our stock." Teva Pharmaceutical Industries Ltd. (TEVA) said Thursday that it plans to reduce 14,000 jobs globally or more than 25% of its total workforce in the next two years. It will immediately suspend dividends on ordinary shares, and cancel bonus for 2017. The company will continue to review the potential for additional divestment of non-core assets. Checkout the few notable companies that are scheduled to release quarterly financial results on Thursday, December 14, 2017. - Growing Cloud Footprint To Bolster ADBE Q4 - COST Q1 Likely To Benefit From Holiday Weekend Sales - Growth And Diversification Strategy To Boost JBL Q1 Results - Will Cloud Propel ORCL Quarterly Performance?
comments powered by Disqus
Follow RTT