BioMarin Pharmaceuticals’ investigational gene therapy valoctocogene roxaparvovec safely and significantly reduced bleeding events — including in target joints — and the use of prophylactic clotting factor VIII in adults with severe hemophilia A, data from a Phase 1/2 study show. These results, along with an interim analysis of…

Recently, I had the pleasure of speaking with Dr. Carol Kasper, a highly regarded hematologist who has dedicated her life’s work — over 40 years of research and patient care — to individuals with hemophilia. Although Kasper retired three years ago, she continues to…

Hemophilia News Today brought you daily coverage of important discoveries, treatment developments, clinical trials, and other key events related to hemophilia throughout 2019. As we look forward to bringing you more news in 2020, we would like to remind you of the 10 most-read stories of 2019. No.

Takeda has presented early data on the prevalence of — and a possible solution for — one of gene therapy’s main hurdles: the development of an immune reaction against the viral-based delivery vectors used in such therapies. The findings, presented at the 61st American Society of Hematology (ASH) Annual Meeting…

I love the holidays — candlelight, decorations, the smell of baking, and time with family. I look forward to the holidays every year, but I am also keenly aware that holidays can be more stressful with the realities of managing hemophilia. Here are some tips for…

To help promote healthy lifestyle changes, the pharmaceutical company Takeda has enrolled some 900 patients with bleeding disorders like hemophilia in the Virgin Pulse Global Challenge. The annual well-being Global Challenge aims to equip teams of employees with the knowledge, tools and support necessary to enhance performance and…

Bayer has entered into a three-year research partnership with the Children’s Hospital of Philadelphia to develop small molecule non-replacement therapies as a first-line and less burdensome oral treatment for hemophilia A and B. Hemophilia is caused by a lack of blood clotting factors or their faulty production,…

The World Federation of Hemophilia states that any person, male or female, with factor levels below 40 percent has hemophilia. However, conventional medical wisdom has not caught up. Women who have abnormal bleeding should be assessed for hemophilia, particularly when they have a family history. Yet too…

New preclinical data on one of Catalyst Biosciences’ lead candidate therapies — marzeptacog alfa activated (MarzAA), an engineered clotting Factor VIIa (FVIIa) given as an under-the-skin injection — support its potential to treat acute bleeds in people with hemophilia and its ability to be used safely in combination with Hemlibra…

Bayer’s investigational gene therapy BAY 2599023 safely and effectively increased the levels of clotting factor VIII (FVIII) and prevented or lessened bleeding in the first two people with severe hemophilia A treated in a Phase 1/2 clinical trial, preliminary data show. The ongoing trial (NCT03588299; 2017-000806-39) is enrolling…