Certainly! Here are some updates on gene therapy for Hemophilia A (factor VIII deficiency) based on the knowledge base:

- Breakthrough Results: A recent gene therapy trial showed promising results for Hemophilia A. Participants saw their factor VIII levels reach or approach normal levels after receiving the therapy. This Show Full Answer

Certainly! Here are some updates on gene therapy for Hemophilia A (factor VIII deficiency) based on the knowledge base:

- Breakthrough Results: A recent gene therapy trial showed promising results for Hemophilia A. Participants saw their factor VIII levels reach or approach normal levels after receiving the therapy. This improvement began a month after the one-time gene therapy and continued over 18 months of follow-up.

- Potential Game-Changer: If these results hold up in larger, longer trials, it could significantly reduce or eliminate the need for regular factor VIII infusions, which are currently required as often as every other day.

- Clinical Trials: In a British trial, all 13 adult patients with Hemophilia A were able to go without preventive injections of a blood-clotting factor that they needed almost weekly. Ten of the 13 patients experienced no bleeding incidents requiring transfusion.

- Safety and Cost: While the trial showed no adverse immune system reactions, researchers caution that it's too soon to draw conclusions about the long-term safety of the treatment. Additionally, gene therapy is expected to be expensive, with costs ranging from $400,000 to $1 million per treatment.

- Expert Opinions: Dr. Suchitra Acharya noted that this strategy could be groundbreaking, potentially freeing patients from bleeding symptoms without the need for clotting protein replacement.

For more detailed information, you can refer to the articles on MyHemophiliaTeam:
- [Another Gene Therapy Breakthrough Against Hemophilia](https://www.MyHemophiliaTeam.com/resources/anot...)
- [Hemophilia Gene Therapy: 7 Things To Consider](https://www.MyHemophiliaTeam.com/resources/hemo...)

 This AI-generated response comes from MyHaemophiliaTeam and other selected sources. It is not a substitute for medical advice. Always ask your doctor about specific health concerns.

Another Gene Therapy Breakthrough Against Hemophilia | MyHemophiliaTeam

Hemophilia Gene Therapy: 7 Things To Consider | MyHemophiliaTeam