A new type of gene-editing treatment being developed by researchers in Japan corrected mutations causing severe hemophilia B in…
Articles by Margarida Maia, PhD
At the World Health Assembly (WHA), member states of the World Health Organization (WHO) unanimously adopted a resolution on…
In the large clinical trial that supported the 2024 approval of Hympavzi (marstacimab) for adults and children with…
The one-time gene therapy BBM-H901 (dalnacogene ponparvovec) has been officially approved in the Macao region of China for the treatment…
Many boys and young men with hemophilia regularly participate in sports. However, doing so frequently — particularly three or…
In boys and men with severe hemophilia A, starting prophylaxis — preventive treatment given regularly to reduce bleeding —…
Testing levels of thrombin — an enzyme that helps blood clot — in people with acquired hemophilia A could…
A single infusion of Hemgenix (etranacogene dezaparvovec-drlb) helped boost levels of factor IX (FIX), the clotting protein missing in…
Altuviiio (efanesoctocog alfa) reduced bleeding and was safe for boys and young men with severe hemophilia A, according to…
A new study highlights a hidden emotional burden for families living with hemophilia: the deep sense of guilt felt…