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Endari: A Treatment Option for Sickle Cell Disease

key information

source: Sickle Options

year: N/A

summary/abstract:

Endari is an amino acid, approved by Food and Drug Administration (FDA) to treat sickle cell disease. It is available in powder form to be taken orally. 

The exact mechanism of action of Endari is not fully understood but it increases the amount of free glutamine in the blood.  Glutamine is used to make anti-oxidant molecules which reduce the oxidative stress in sickle red blood cells resulting in increased flexibility of red blood cells while they travel through the body. 

Safety profile of Endari is not available for children less than 5 years of age and risk of taking Endari in pregnancy is not known yet.

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