Can gene therapy help my teen son with hemophilia?

Question

Hello doctor,

My son is 12 years old and has hemophilia A. We discovered it when he had excessive bleeding after a minor injury. We are managing with factor replacement therapy, but sometimes the medicines are not readily available, and the costs are very high. I am worried about his future.

Will he always need these injections?
Can gene therapy cure hemophilia, and is it available in India?
Also, how can we keep him safe during school activities without making him feel different from others?

Please help.

Thank you.

Answer 1

Hello,

Welcome to icliniq.com.

I have gone through your query and understand your concern.

Hemophilia A is a lifelong condition caused by a deficiency of clotting factor VIII, so factor replacement therapy remains the mainstay of treatment to control bleeding and prevent complications. However, it can be costly and sometimes difficult to access.

In recent years, new options such as Emicizumab, a subcutaneous injection given less frequently, have reduced the burden of regular factor infusions for many children. Gene therapy has also shown promising results in clinical trials, offering the possibility of long-term or even near-permanent correction by introducing a functional factor VIII gene. It is gradually becoming available in certain countries, including some centers in India, though access and affordability remain challenges.

Until such options are more widely accessible, consistent factor prophylaxis and preventive care are most important. At school, it is best to inform teachers discreetly so they know how to respond in case of bleeding while allowing your son to participate safely. Low-impact activities like swimming, walking, and certain non-contact sports are usually encouraged, while high-contact sports should be avoided.

Helping him feel included while ensuring safety is key, and many children with hemophilia can lead active, fulfilling lives with proper care.

I hope I have answered your question.

Let me know if I can assist you further.

Thank you.