• Join Today!

Become a member and connect with:

  • An Active Online Community
  • Articles and Advice on SCD
  • Help Understanding Clinical Trials
scientific articles

Sickle cell therapy drought ends

key information

source: JAMA

year: 2017

authors: Voelker R

summary/abstract:

After nearly 20 years without new treatments for sickle cell disease, the FDA has approved L-glutamine oral powder to reduce severe complications of the blood disorder in patients aged 5 years or older. It also is the first sickle cell disease treatment approved for pediatric patients.

Marketed as Endari, L-glutamine is an amino acid that can help prevent sickle red blood cells from sticking to the endothelial cells of blood vessels, which causes vaso-occlusion that results in severe pain and organ damage. Previously, the antimetabolite hydroxyurea had been the only FDA-approved drug for sickle cell disease.

DOI: 10.1001/jama.2017.10152

read more full text source

expertly curated content related to this topic

To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More information

The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.

Close