Author Archives: Steve Bryson PhD

Patients on Prophylactics Maintain Pain Thresholds Over Year Study

Differences in sensory detection and pain thresholds were found in hemophilia patients taking prophylactics, compared to healthy individuals, and these differences did not change over the course of a one-year follow-up study. These findings indicate that “under prophylactic treatment, the existing difference in the pain profile between [people with…

FDA Puts Hold on SIG-001 Phase 1/2 Trial for Hemophilia A

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on Sigilon Therapeutics’ Phase 1/2 study evaluating the investigational therapy SIG-001 in adults with severe or moderately severe hemophilia A. The move comes after a serious adverse event (SAE) occurred involving one of the three participants…

Gene Therapy Roctavian Prevents Bleeds Over 5 Years, Data Show

The investigational gene therapy Roctavian continues to effectively and safely prevent bleeding episodes and the need for clotting factor VIII replacement therapy in adults with severe hemophilia A, five-year data from a Phase 1/2 study show. BioMarin Pharmaceutical, the therapy’s developer, plans to share the data in…

First Patient Dosed in Open-label Trial of MarzAA

The first participant has been dosed in a Phase 1/2 clinical trial evaluating marzeptacog alfa activated (MarzAA) to treat bleeding episodes in patients with factor VII (FVII) deficiency, Glanzmann thrombasthenia, and hemophilia A with inhibitors receiving treatment…

Scientists Target FVIII Inhibitor Formation in Hemophilia A

Scientists found the immune signaling protein BAFF can promote the formation of neutralizing antibodies, or inhibitors, against blood clotting factor VIII (FVIII), which can lower the effectiveness of FVIII replacement therapies used to treat people with hemophilia A.  They also discovered that hemophilia A…