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…
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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…
While there is no cure for hemophilia, it is very possible to live with this genetic bleeding disorder and maintain a good quality of life. Proper nutrition and weight management combined with a safe and effective exercise plan monitored by a medical professional can make…
Switching to extended half-life (EHL) products has provided a reduction in the number of infusions and fewer bleeding events compared to standard treatments, particularly in hemophilia B, according to early real-world data from Europe. The study, “Real‐life experience in switching to new extended half‐life products…
Patrice Thomas has utilized her skills as a women’s healthcare advocate to learn about marginalized communities and support structural change. Through her current position at the Hemophilia Foundation of Michigan (HFM), Patrice is bringing together women with hemophilia and healthcare providers to educate and break down barriers…
Pfizer’s Expansion in North Carolina May Help Advance Manufacturing of Hemophilia Gene Therapies
Pfizer will invest an additional $500 million into the construction of its new manufacturing facility in Sanford, North Carolina, the company announced. According to the company, the facility could help advance the manufacturing of one-time gene therapies that use modified adeno-associated viral vectors, such as SB-525, which is…
Mild to moderate hemophilia B affects the relationships of adult patients, children with the disease and their caregivers in aspects such as telling others about the diagnosis, selecting a partner, and feeling bullied, according to an American study. The research, “Mild-severe hemophilia B impacts relationships of…
Although most patients report that discussing their sexual health with their doctors is helpful, hemophilia significantly affects the quality of their sex lives, according to the results of two studies. The research, “Hemophilia and sexual health: results from the HERO and B-HERO-S studies,” was published in…
The prevalence of hypertension is higher in patients with hemophilia than in the general population, according to a nationwide study in Sweden with long-term follow-up. In contrast, no differences were seen in the prevalence of angina pectoris and myocardial ischemia, two cardiovascular diseases. The study, “…