logo
Plus   Neg
Share
Email
Comment

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
BlackRock Inc., the world's largest asset manager, said it plans to speak with gun makers and distributors following public outcry after the Florida high school shooting that killed 17 people. BlackRock, which had $6.3 trillion in assets under management as of December 31, 2017, holds shares in gun makers Sturm Ruger & Co. Inc. as well as American Outdoor Brands Corp. An upgraded boarding in Southwest Airline will cost you more. The airline usually not assign seats to passengers, but one can choose from an open seat. The Upgraded Boarding will allow passengers to choose from A1 - A15 boarding positions. The cost for these positions would be $30, $40 and $50, depending on flight and route. This option can be availed from the ticket counter or gate. Citigroup Inc.'s co-head of mergers and acquisitions, Peter Tague, is leaving the company, according to media reports, citing people familiar with the matter. Tague has been co-head of Global M&A business at Citigroup since March 2012, alongside Cary Kochman and Mark Shafir. It was not immediately clear what Tague intends to do after he leaves Citigroup.
comments powered by Disqus
Follow RTT