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Novel Sickle Cell Drug Causes Radical Results in End of Life Patients

This week the FDA approved a novel treatment for sickle cell disease, a serious and sometimes deadly disease that affects around 100,000 Americans. The treatment was found to exceptionally improve the health and quality of life in the “end of life” patients involved in the trials, and experts believe that upon release, it may do much to improve the lives of sickle cell patients throughout the world.

Oxbryta is an inhibitor of deoxygenated sickle hemoglobin polymerization and was granted Accelerated Approval. This classification allows the FDA to quickly approve drugs for serious conditions. The drug’s trial consisted of 274 patients with sickle cell disease who received different doses of the medication or a placebo, a press release explained.

Results showed that the medication was able to increase the hemoglobin response rate in patients far more than those in the placebo group. This is important as sickle cell affects the hemoglobin, a type of red blood cell, and leads to some of the disease’s devastating effects.