A first patient has been dosed in XTEND-Kids, a Phase 3 clinical trial evaluating the safety and efficacy of the investigational replacement therapy efanesoctocog alfa in boys with severe hemophilia A. Hemophilia A is caused by the lack of a functional blood clotting protein called factor VIII…

The paladin By-Tor from the Order of Light fights to protect others and always chooses life over death, even when it places him in certain danger. In the world of Dungeons and Dragons, he uses his sword and…

Long-term treatment with Jivi safely and effectively reduced the number of bleeding episodes in adolescents and adults with severe hemophilia A, according to up to seven years of data from the Phase 2/3 PROTECT VIII trial and its extension study. The new data confirmed the previous findings from…

From donning red gear to flooding social media, supporters are gearing up for World Hemophilia Day 2021 on April 17. The event is focused on raising awareness and understanding of the bleeding disorder that affects about one in 10,000 people globally. Organized by the World Federation of Hemophilia (WFH),…

I live in the middle of nowhere. It is one of the most beautiful places on the planet. The forest and the ocean intersect, creating some of the most gorgeous hiking trails I have had the pleasure of traversing. Nature is our playground, with thousands of acres of beaches, forests,…

Women who are hemophilia carriers are at a higher risk of experiencing heavy bleeding after giving birth, particularly those who are not on a prophylactic, or preventive, treatment regimen, a new review study has found. Researchers say these findings indicate that more intensive preventive treatments might benefit carriers about…

The World Federation of Hemophilia (WFH) announced that Takeda has joined its WFH Humanitarian Aid Program, agreeing to large, annual donations of treatments for people with inherited bleeding disorders like hemophilia in developing countries. Takeda,…

AMT-061 (etranacogene dezaparvovec), uniQure’s investigational gene therapy for hemophilia B, likely did not cause the case of hepatocellular carcinoma (HCC) — a form of liver cancer — that led to the program’s clinical hold in December 2020, an investigation has found. The U.S. Food and Drug Administration (FDA) had put the AMT-061…

Jivi was safe and effective at preventing and treating bleeds for more than five years in boys under age 12 with severe hemophilia A, according to long-term data from a Phase 3 trial’s extension study. Data analysis also showed that most of…

Sometimes I feel like I don’t belong. I’m a misfit, an imposter. I feel fine and haven’t had a bleed in almost a month. Do I really have hemophilia? Do I really need to infuse every three…