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Sickle Cell Anemia Therapeutics

Sickle cell disease (SCD) is a group of inherited red blood cell disorders that cause red blood cells to become misshapen and break down. It is caused by a problem in the hemoglobin-beta gene found on chromosome 11. Treatments include medication, blood transfusions, and rarely a bone-marrow transplant. Although there is no cure for most people with sickle cell anemia, treatments can relieve pain and help prevent complications associated with the disease. People with SCD can live full lives and enjoy most of the activities that other people do.

Sickle cell anemia therapeutics include medication, blood transfusions, and sometimes a bone-marrow transplant. Sickle-cell diseases are types of blood disorders which are usually inherited genetically. Currently, hematopoietic stem cell transplant is the only curative therapy available for patients with sickle cell disease. However, hematopoietic stem cell transplant is a rigorous, time-consuming, potentially high-risk procedure; only used when the benefits of cure outweigh the considerable risks of the procedure, and only when a suitable donor is available.

One of the common types of sickle cell diseases is the sickle cell anemia. Sickle cell anemia is one of a group of inherited disorders known as sickle cell disease. It affects the shape of red blood cells, which carry oxygen to all parts of the body. The cause of SCD is a defective gene, called a sickle cell gene. People with the disease are born with two sickle cell genes, one from each parent. Mild sickle cell disease may have no impact on a person's day-to-day life. But the illness can be serious enough to have a significant effect on a person's life.

It can lead to health problems like strokes, serious infections and lung problems, which can occasionally be fatal. Sickle cell anemia therapeutics are usually aimed at avoiding episodes of pain, relieving symptoms, and preventing complications. Although SCD is common in endemic areas of malaria, it is one of the rare diseases in Japan. SCD is the most common monogenic disorder globally but qualifies as a rare disease in Germany.