Preclinical data bring human trial launch closer for MGX-001
Data from non-human primates suggest gene-editing therapy MGX-001 may be able to restore clinically meaningful levels of factor VIII (FVIII) after…
Articles by Patricia Inácio, PhD
Joint damage common in Chilean children with severe hemophilia
Half of the children in Chile with severe hemophilia A and B exhibit joint damage despite receiving preventive treatment…
Eloctate, Alprolix control bleeds in hemophilia patients’ major surgeries
Treatment with the extended half-life (EHL) therapies Eloctate (efmoroctocog alfa) and Alprolix (eftrenonacog alfa) was found to safely…
Hemophilia B gene therapy Hemgenix to be covered in Canada
The cost of Hemgenix (etranacogene dezaparvovec), a one-time hemophilia B gene therapy, will be covered for patients living…
Little genetic testing for girls at risk of hemophilia carrier status: Study
A single-center U.S. study found relatively little genetic testing was done for girls with a 50% risk of being a…
Blood group O not tied to inhibitor risk in children with severe hem A
Children with severe hemophilia A with blood group O are not at increased risk of developing neutralizing antibodies against…
Saliva protects against mouth and throat bleeds in hemophilia A: Study
Small vesicles in the saliva of people with severe hemophilia A may explain why these patients rarely have bleeds…
2 new US patients given hemophilia B gene therapy Hemgenix: Reports
Two new patients in the U.S. have received Hemgenix (etranacogene dezaparvovec), the first gene therapy to be approved for hemophilia…
Phase 1/2 clinical trial of BE-101 now enrolling hemophilia B patients
The first-in-human Phase 1/2 clinical trial testing BE-101, Be Biopharma’s B-cell treatment candidate for hemophilia B, is now…
Bypassing agent Sevenfact reduces bleeds in patients with inhibitors
Sevenfact (eptacog beta) was deemed safe and effective at stopping bleeds in patients with hemophilia A or B with…