According to the department, the drug is approved for the treatment of patients 12 years and older.
It is specified that the drug is intended for the treatment of sickle cell anemia. It is a group of inherited blood disorders that affect approximately 100,000 people in the United States.
The main problem of this disease is a mutation of hemoglobin, as a result of which red blood cells take on the shape of a crescent or sickle, which leads to problems with the delivery of oxygen to the tissues of the body. This, in turn, leads to severe pain and organ damage and, as a result, can cause disability or even early death.
The decision to register gene therapy was made on the basis of data from studies involving 44 patients with the corresponding disease.
Earlier, the Danish bank Saxo Bank published an annual series of "shocking forecasts". As part of one of the forecasts, a financial institution predicted the emergence of an anti-obesity drug.