A four-year-old Pakistani girl with severe thalassemia has become the first foreign child to be successfully treated with a Chinese-developed gene-editing drug.
Her condition improved dramatically after she received the treatment earlier this year. She is no longer dependent on blood transfusions and has returned to a normal life.
Previously, she required regular transfusions to survive.
A small ceremony was held on Tuesday to mark her recovery.
Earlier this year, Aiza was taken to Shanghai to undergo an experimental gene-editing therapy called CS-101—a base-editing drug developed to treat this disease.
Aiza’s father, Muhammad Adeel—a 40-year-old physicist and former postdoctoral researcher in Hong Kong—expressed gratitude at the event, offering flowers and a thank-you card to the medical team.
He shared that the family received not only exceptional medical care but also strong emotional support throughout the process. Adeel added that her daughter went through emotional ups and downs during recovery, but the healthcare team stood by them the whole way.
What is Thalassemia ?
Thalassemia is an inherited blood disorder that affects the body’s ability to produce haemoglobin and healthy red blood cells. Symptoms resemble those of anaemia and can range from mild to severe. Treatment often includes blood transfusions and iron chelation therapy.
A bone marrow transplant from a compatible sibling offers the best chance at a cure. Unfortunately, most people with thalassemia lack a suitable sibling donor. Also, a bone marrow transplant is a high-risk procedure that may result in severe complications.
You should expect a normal life expectancy if you have mild thalassemia. Even if your condition is moderate or severe, you have a good chance of long-term survival if you follow your treatment program (transfusions and iron chelation therapy).
