The European Commission has conditionally approved Hemgenix (etranacogene dezaparvovec), a one-time gene therapy to treat adults with hemophilia…
Articles by Steve Bryson, PhD
The European Commission has approved Hemlibra (emicizumab) as a routine preventive treatment for people with moderate hemophilia A…
The U.S. Food and Drug Administration (FDA) has cleared CRYOcheck Chromogenic Factor IX assay, a test intended to be…
A review is continuing into a resubmitted application for approval of Roctavian (valoctocogene roxaparvovec), a gene therapy for adults with…
Centessa Pharmaceuticals is set to open PRESent-5, an observational feeder study of hemophilia patients who could then…
Health Canada has expanded its approval of Rebinyn (nonacog beta pegol) as a routine preventive treatment for bleeding episodes…
BioMarin Pharmaceutical has resubmitted an application to the U.S. Food and Drug Administration (FDA) seeking approval of Roctavian…
A significant proportion of bleeds in people with hemophilia A are left untreated, according to an analysis of data…
If approved for hemophilia A, the one-time gene therapy Roctavian (valoctocogene roxaparvovec) would provide substantial cost savings per patient…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to SerpinPC for treating hemophilia B. Orphan…