Top 10 hemophilia stories of 2024
In 2024, Hemophilia News Today provided readers with up-to-date coverage on research, treatments, and clinical trials for hemophilia.
Articles by Margarida Maia, PhD
Altuviiio bests others as hemophilia A factor replacement therapy
For adults and adolescents with hemophilia A without inhibitors, Altuviiio (efanesoctocog alfa) may work better than other standard…
Concizumab reduces bleeding in patients without inhibitors
Adults and adolescents with hemophilia A or hemophilia B without inhibitors experienced significantly fewer bleeding episodes when using…
Hympavzi approved in EU for hemophilia A, B, without inhibitors
Pfizer’s Hympavzi (marstacimab) has been approved in the European Union (EU) to prevent or reduce the frequency of bleeding…
Hympavzi may be available as prophylaxis in US before year’s end
Hympavzi (marstacimab-hncq) may be available by prescription in the U.S. before the end of the year. The therapy was…
FDA approves marstacimab, now Hympavzi, for hemophilia
The U.S. Food and Drug Administration (FDA) has approved Pfizer’s marstacimab under the brand name Hympavzi for use as…
Hemophilia gene therapy Beqvez reduces bleeds in Phase 3 trial
Hemophilia gene therapy Beqvez (fidanacogene elaparvovec), marketed as Durveqtix in the European Union, results in stable production of factor IX…
Joint problems in hemophilia A are common despite prophylaxis
Almost two-thirds of men with moderate or severe hemophilia A have joint problems that cause pain and reduce their…
Eloctate as prophylaxis eases pain due to hem A, improves life quality
Long-term prophylaxis or preventive treatment with Eloctate (efmoroctocog alfa) helps to ease pain and improve health-related life quality for…
Roctavian nearly zeroes out bleeds, prophylaxis need over 7 years
A single dose of Roctavian (valoctocogene roxaparvovec-rvox) nearly zeroed out the number of bleeds and the need for prophylaxis,…