Potential hemophilia A gene therapy SPK-8011 moves to Phase 3
Roche is initiating a Phase 3 clinical trial to test the experimental gene therapy SPK-8011 in people with…
Articles by Marisa Wexler, MS
Nuwiq prevents and controls bleeds in patients new to FVIII therapy
Nuwiq (simoctocog alfa) effectively prevented and controlled bleeds in previously untreated people with severe hemophilia A, according to…
Orsini pharmacy selected to supply Roctavian for hemophilia A patients
Orsini Specialty Pharmacy has been selected to distribute the new gene therapy Roctavian (valoctocogene roxaparvovec-rvox) for use by…
No bleeds seen over 1 year on Altuviiio for many Hem A children
Nearly two-thirds of children with severe hemophilia A given once-weekly Altuviiio (efanesoctocog alfa) in a Phase 3…
Hemgenix given to treat first hemophilia B patient in US
A first adult with hemophilia BÂ in the U.S. has been treated with commercially available Hemgenix (etranacogene dezaparvovec), a…
Ixinity up for FDA approval for children under 12 with hemophilia B
The U.S. Food and Drug Administration (FDA) has agreed to review Medexus Pharmaceuticals’ request to approve Ixinity (trenonacog…
Better outcomes seen with early preventive hemophilia A treatment
People with hemophilia A who start on preventive treatment in the first years of life generally report better health-related…
AstraZeneca, Sernova join forces on cell therapy research
Note: This story was updated May 8, 2023, to remove hemophilia as one of the specific indications the collaboration will…
Concizumab approved in Canada for hemophilia B with inhibitors
Concizumab has been approved as a preventive treatment in Canada for hemophilia B patients ages 12 and older who…
Tardigrade proteins may aid stability, duration of treatments
Proteins from tardigrades — microscopic animals known for their ability to survive in extreme conditions — could be used to…