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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

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