Sickle cell therapy drought ends

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. Abstract source Full text