The parents of most children with severe hemophilia A who took part in the XTEND-Kids clinical trial of…
Articles by Marisa Wexler, MS
The once-weekly injection therapy marstacimab is being considered for approval in both the U.S. and Europe for people with…
A first patient has been dosed in a Phase 1/2a clinical trial that’s assessing the safety and preliminary efficacy of…
AAV5 DetectCDx, a companion diagnostic test used to determine patient eligibility for the hemophilia A gene therapy Roctavian…
The first participant has been dosed in a Phase 2b clinical trial that’s assessing the safety and efficacy of SerpinPC…
Treatment with platelets stripped of their natural sugar molecule coating helped to prevent inhibitors, or neutralizing antibodies, from forming against…
Altuviiio (efanesoctocog alfa) has been approved in Japan and Taiwan as a treatment to prevent and control bleeding in…
Treatment with Idelvion (albutrepenonacog alfa) was effective in a real-world setting at controlling bleeds for five people with…
The U.S. Food and Drug Administration (FDA) is considering updating the prescribing information for Altuviiio (efanesoctocog alfa) to include…
To help advance new treatments for hemophilia and other blood disorders, Pathway to Cures (P2C) has invested $250,000…