Updated Label for Endari, A Sickle Cell Disease Treatment

The FDA has recently approved of an updated label for Endari, a treatment for sickle cell disease (SCD). This new label will give medical professionals better information, allowing them to make more informed treatment decisions for patients.

About Endari

Endari is prescribed to reduce complications from SCD in patients over the age of five. It was first approved back in July of 2017, but sales did not begin until 2018. The new label contains information on Endari in combination with hydroxyurea. It has already been able to improve the lives of patients, but now that it has an updated label, it will be more effective.

About Sickle Cell Disease (SCD)

Sickle cell disease (SCD) includes a group of disorders that are characterized by malformed red blood cells that look like sickles. The most common and severe form of this disease is sickle cell anemia. In sickle cell disease, deformed cells cause blockages and restricted blood flow as they get caught along the walls of blood vessels. This disease is caused by a mutation in the gene responsible for producing hemoglobin, which carries oxygen throughout the body. This gene is inherited in an autosomal recessive pattern, meaning both parents must pass down a copy to their child. Sickle cell disease is most common in people of African and Hispanic descent.

Symptoms of sickle cell disease include pain crisis, swelling of the hands and feet, and symptoms associated with anemia like fatigue, jaundice, and delayed growth. Adults tend to constantly feel the effects of this disease, but children usually only experience them during pain crisis. Regardless of age, damage usually occurs to the organs that are affected by the blocked blood flow. The most commonly damaged organs are the brain, eyes, spleen, liver, kidneys, lungs, heart, skin, joints, and bones. There is no cure for sickle cell disease. While some people qualify for bone marrow and blood transplants, not everyone is eligible for this procedure. Other forms of treatment are symptomatic and meant to prolong life.

Find the source article here.

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