The US Food and Drug Administration (FDA) has approved gene therapy for the first time in the treatment of sickle cell disease (SCD) patients aged 12 or older. In this case, the treatment of SCD sufferers is arranged through Casgevi and Livgenia treatment process.
SCD is a blood disorder. Millions of people in America are affected by this disease. Most of them are Afro-American. In addition, many Hispanic Americans also suffer from this disease. In this case the red blood cells are crescent shaped due to mutation in hemoglobin. These red blood cells block blood flow. Oxygen supply to body tissues is also affected. It causes severe pain in the body. Limbs are also affected. Patients with SCD are often in a critical condition. Many die at a young age.
SCD is a rare disease, said Nicole Vardan, director of the FDA’s Office of Therapeutic Products. Gene therapy, which has been approved by the FDA, is very important in the treatment of this disease.
Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said the approval for the treatment of SCD is a medical milestone. This approval reflects advances in the medical system. This subject has undergone repeated experimental applications prior to FDA approval. To ensure safety and effective treatment of SCD sufferers.
44 patients were treated with Casgavi. 31 of them are monitored at regular intervals. 29 of them have been successfully treated. It should also be noted that none of the patients had adverse effects of this treatment.
Lifgenia is also being tested for 24 months in patients with SCD aged 12 to 50 years. In this case, 28 out of 32 patients were successful in experimental application.
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