The New England Journal of Medicine
e-mail icon  FREE NEJM E-TOC    HOME   |   SUBSCRIBE   |   CURRENT ISSUE   |   PAST ISSUES   |   COLLECTIONS   |    Advanced Search
Sign in | Get NEJM's E-Mail Table of Contents — Free | Subscribe
 
Editorial
PreviousPrevious
Volume 358:2829-2831 June 26, 2008 Number 26
NextNext

A Small Molecule for a Large Disease
Reed E. Pyeritz, M.D., Ph.D.

Since this article has no abstract, we have provided an extract of the first 100 words of the full text and any section headings.

 Sign up for free e-toc
 

This Article
-Full Text
- PDF
-PDA Full Text
-Purchase this article

Tools and Services
-Add to Personal Archive
-Add to Citation Manager
-Notify a Friend
-E-mail When Cited
-E-mail When Letters Appear

More Information
-Related Article
 by Brooke, B. S.
-PubMed Citation
Almost three decades ago, Victor McKusick and I reviewed Marfan's syndrome in the Journal and advised traditional medical and surgical approaches to management.1 The holy grail at that time was the cause of this autosomal dominant condition, based on the common presumption that understanding the cause would lead directly to effective therapy. In fact, life expectancy improved dramatically in subsequent years for all but the most severely affected patients, despite a lack of understanding of the underlying connective-tissue defect.2 Dissection of the aorta was and remains the most common cause of death. However, the evolution of ever more effective surgical . . . [Full Text of this Article]


Source Information

From the Center for the Integration of Genetic Healthcare Technologies, Departments of Medicine and Genetics, University of Pennsylvania School of Medicine, Philadelphia.




HOME  |  SUBSCRIBE  |  SEARCH  |  CURRENT ISSUE  |  PAST ISSUES  |  COLLECTIONS  |  PRIVACY  |  HELP  |  beta.nejm.org

Comments and questions? Please contact us.

The New England Journal of Medicine is owned, published, and copyrighted © 2008 Massachusetts Medical Society. All rights reserved.