A gene therapy approach using a so-called antibody-drug conjugate (ADC) conditioning regimen led to safe and sustained production of factor VIII (FVIII) in platelets, and prevented joint bleeding in a mouse model of hemophilia A, according to new research. The study, “Nongenotoxic antibody-drug conjugate conditioning…

Intimacy. Warmth. Comfort. Security. Closeness. These are things that many of us yearn for in relationships. However, when someone has a bleeding disorder, relationships can also be associated with other things, like fear, pain, embarrassment, and shame. Sexuality is hard to discuss,…

Receiving a hemophilia diagnosis can leave patients and their caregivers with many unanswered questions about the disease. Many want to know how to find information, connect with other patients and families, and get support. Here are answers to some frequently asked questions about hemophilia: What is hemophilia?…

Using Hemlibra (emicizumab) as prophylaxis lowers treatment costs considerably for people with hemophilia A compared to preventative factor (FVIII) treatment, according to an economic model study. The model predicts $47,814 in savings to U.S. payers in year one, and a potential for more $3 billion in savings over 20 years…

Many hemophilia patients experience undiagnosed symptoms of depression and anxiety that can impact their ability to cope with the disease and to adhere to treatment, a U.S. survey says. These findings shed light on psychological factors that affect the course of the disease, but with dedicated management and follow-up, depression…

Hemlibra (emicizumab) is safe and effective to treat children younger than 12 who have hemophilia type A and are negative for antibodies against synthetic factor VIII, results from a Japanese clinical study show. The trial findings were reported in the study, “A multicentre, open‐label study of emicizumab given…

Last week, I read Gill Paul’s latest novel, “The Lost Daughter.” The book is a historical fiction account of Russia’s Romanov family. It is well-known that the Romanovs and hemophilia are connected, but I had no expectation that bleeding would play into the plot. Near the beginning of…

Data from the first two patients treated with FLT180a, Freeline’s single-infusion gene therapy for patients with hemophilia B, showed a rise in levels of clotting factor IX to 40%, with levels remaining stable for over a year. Pratima Chowdary, principal investigator for the FLT180a program, presented the data…

Scientists report that hemophilia is three times more prevalent in men worldwide than previously thought and still associated with a poorer life expectancy, especially in low-income countries. Findings of the study, “Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males: A Meta-analytic Approach Using National Registries,”…

When you look at me, you’ll see a quirky, colorful, loud, and active person, yet at the same time, I am reserved. I am of average height and weight, and am known as a hiker, a cyclist, and (for very short stints) a beach jogger. I smile a…