New article published: Hermans et al, 2025

Congratulations to Dr. Van Thillo and Prof. Hermans on the publication of their review article on rebalancing agents in hemophilia in the journal Haemophilia. We are delighted to have been able to contribute to the success of this article!

Hemophilia is a 𝐠𝐞𝐧𝐞𝐭𝐢𝐜 𝐝𝐢𝐬𝐨𝐫𝐝𝐞𝐫 that causes abnormal blood clotting. There are 𝐬𝐞𝐯𝐞𝐫𝐚𝐥 𝐭𝐲𝐩𝐞𝐬 𝐨𝐟 𝐡𝐞𝐦𝐨𝐩𝐡𝐢𝐥𝐢𝐚, the two main ones being 𝐡𝐞𝐦𝐨𝐩𝐡𝐢𝐥𝐢𝐚 𝐀, caused by a deficiency of clotting factor VIII, and 𝐡𝐞𝐦𝐨𝐩𝐡𝐢𝐥𝐢𝐚 𝐁, caused by a deficiency of factor IX. As the genes for these clotting factors are located on the X chromosome, women are 𝐥𝐞𝐬𝐬 𝐜𝐨𝐦𝐦𝐨𝐧𝐥𝐲 𝐚𝐟𝐟𝐞𝐜𝐭𝐞𝐝, while boys are always affected if they inherit a mutated gene. The severity of the condition varies, 𝐫𝐚𝐧𝐠𝐢𝐧𝐠 𝐟𝐫𝐨𝐦 𝐦𝐨𝐝𝐞𝐫𝐚𝐭𝐞 𝐭𝐨 𝐬𝐞𝐯𝐞𝐫𝐞.

There are 𝐬𝐞𝐯𝐞𝐫𝐚𝐥 𝐭𝐲𝐩𝐞𝐬 𝐨𝐟 𝐭𝐫𝐞𝐚𝐭𝐦𝐞𝐧𝐭. 𝐑𝐞𝐩𝐥𝐚𝐜𝐞𝐦𝐞𝐧𝐭 𝐭𝐡𝐞𝐫𝐚𝐩𝐢𝐞𝐬 restore coagulation by replacing the dysfunctional factor. 𝐁𝐲𝐩𝐚𝐬𝐬𝐢𝐧𝐠 𝐚𝐠𝐞𝐧𝐭𝐬 mimic the action of coagulation factors. 𝐆𝐞𝐧𝐞 𝐭𝐡𝐞𝐫𝐚𝐩𝐲 is another therapeutic option for adults who do not produce antibodies against the dysfunctional factor. In this review, the authors focused on a 𝐧𝐞𝐰 𝐜𝐥𝐚𝐬𝐬 𝐨𝐟 𝐫𝐞𝐛𝐚𝐥𝐚𝐧𝐜𝐢𝐧𝐠 𝐚𝐠𝐞𝐧𝐭𝐬 that inhibit various anticoagulants, such as fitusiran, concizumab, marstacimab, serpinPC, and VGA039.

These treatments are promising, but 𝐬𝐞𝐯𝐞𝐫𝐚𝐥 𝐪𝐮𝐞𝐬𝐭𝐢𝐨𝐧𝐬 𝐫𝐞𝐦𝐚𝐢𝐧 𝐭𝐨 𝐛𝐞 𝐬𝐭𝐮𝐝𝐢𝐞𝐝. The article is available on the journal's website. Doi: 𝟏𝟎.𝟑𝟑𝟐𝟒/𝐡𝐚𝐞𝐦𝐚𝐭𝐨𝐥.𝟐𝟎𝟐𝟓.𝟐𝟖𝟖𝟐𝟒𝟓.