Gene Therapy for Hemophilia: A cure for the Royal disease?

Hemophilia is a severe bleeding disorder caused by a deficiency of Factor VIII or IX. Patients are treated with intravenously administered coagulation factor concentrate 2-4 times every week to prevent severe and frequent bleeding episodes
AAV-based gene therapy aims to increase factor levels in order to reduce the risk of bleeding by expression of FVIII and IX in the liver. In the Erasmus MC 6 patients have successfully been treated in the last few years, leading to reduced bleeding without the need of factor concentrates.
In this webinar we will report the latest results on efficacy, side effects and hurdles of gene therapy. Given the increased number of gene therapy initiatives for other rare diseases, the Erasmus MC should invest in gene therapy in order to cure rare inherited disorders and improve patient outcome and quality of life.

[@bhaibhaitelecombhaitelecom7201]

Congretchulation

[@shreejalk.c.4630]

From Nepal ❤

[@SaChin-sc1tp]

could it just be that the artificial factor 8 produced by the virus is interfering with the liver and causing the elevated levels of ALT?
could we have an improved gene therapy in the near future without THE elevated ALT levels preventing liver damage and therefore no need for steroids since they have bad side effects?

[@bhaibhaitelecombhaitelecom7201]

I am haemophilia patient in Bangladesh, dear doctor hurryup factor 8 cure

[@ptrreddy3132]

i am hemophilia patient when come gene therapy india

[@erasmusmc]

Hi, thank you for your question. I recommend you to contact the Hemophilia Treatment Center by calling (010) 704 01 13. ^Team Erasmus MC

[@Mahimfarazi13]

Gene editing could be cured hemophilia A... Pls go on trial for hemophilia A, gene editing soon f8

[@newtechbymj42161]

😭 hemophilia

[@xboy2374]

gene tharepy