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FDA Gives Approval to Endari to Treat Sickle Cell Disease

By jeremyc | July 16, 2017

The FDA has given its approval to Endari for reducing serious complications linked to sickle disease in patients aged 5 years or more. Sickle Disease is a term used to describe a group of genetic red blood disorders. Sickle disease patients have abnormal hemoglobin, a protein responsible for carrying oxygen all across the body, in the red blood cells.

This is a genetic or inherited condition. That is, the patient gets it from his or her parents. In sickle disease patients, the red blood cells are shaped abnormally.

Normal red blood cells are shaped like a disc. Thanks to this shape, the red blood cells are able to comfortably move through small and large blood vessels and effectively deliver oxygen. However, in case of patients with sickle disease, the red blood cells have a shape similar to a sickle. Because of this abnormal shape, the red blood cells are not able to move freely in blood vessels. This in turn restricts the delivery of oxygen to tissues, causing sharp pain and damage to organs.

The last treatment for sickle disease that FDA approved was 20 years back. Till now there was only one drug available for sickle disease. Now with the approval of this new drug, the patients have one more treatment option.

Like other drugs, Endari might cause side effects. Common side effects associated with Endari use are nausea, constipation, abdominal pain, back pain, chest pain, headache, and cough.

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