Pediatrics

Genetically Engineered Enzyme Boosts Treatment for Rare Children's Disease

Published in Gene Therapy Weekly, February 8th, 2001

U.S. medical scientists, working together, have found that a genetically engineered enzyme can significantly help children and young adults with a rare disorder that involves storage of materials within cells, resulting in the cells swelling to the point of bursting and killing those cells.

In its most severe form, the disease, mucopolysaccharidosis 1, causes progressive developmental delays, corneal clouding, lung obstructions, heart disease, severe joint stiffness, and death, usually by age 10. In milder forms, known as Hurler-Scheie syndrome and Scheie syndrome, patients suffer many of the same medical problems, but they develop more slowly, and patients die by...

Want to see the full article?

Purchase this article for only $3.00

We're a pay-per-view site for premium content. If you'd like to purchase this article, it's only $3.00.

Welcome to NewsRx!

Learn more about a six-week, no-risk free trial of Gene Therapy Weekly

Source: Gene Therapy Weekly (2001-02-08)

NewsRx is Social

Top Medical Newsletters

Awards

Best Health/Healthcare Content, 2012
Best Health/Healthcare Content, 2011
Best e-Business Site, 2010
Best e-Business Site, 2009
Best e-Business Site, 2008
Best e-Business Site, 2007
Best e-Business Site, 2006
Best Healthcare Content, 2005
Best Overall Internet Site, 2005
Best Interactive Site, 2005

Facts & Stats

NewsRx also is available at LexisNexis, Gale, ProQuest, Factiva, Dialog, Thomson Reuters, NewsEdge, and Dow Jones.

Google 2010 PageRank: #2 Among Top Health News and Media Publications
Google 2010 PageRank: #2 Among Top Science Publications in Biology/Physiology
Google 2010 PageRank: #2 Among Top News and Media for the Business of Pharmaceuticals
Amazon's Alexa 2010 PageRank: #2 News and Media Site for the Pharmaceutical Industry