News

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,”…

Combining immune tolerance induction, used to prevent the development of anti-factor VIII inhibitors, with Hemlibra (emicizumab) is a feasible and safe way of treating children with severe hemophilia A, a study shows. Published in the journal Haemophilia, the report covers clinical outcomes in the first seven patients to be…

Hemophilia A patients undergoing total hip or knee replacement/reconstruction surgeries have lesser blood loss and require a lower transfusion amount if treated with Cyklokapron (tranexamic acid), a study reports. They also are likely to experience less joint pain or swelling and better joint function, and have lower levels…

uniQure has achieved its target patient enrollment goal ahead of schedule for its ongoing Phase 3 HOPE-B trial. The trial is assessing the safety and efficacy of AMT-061 (etranacogene dezaparvovec), the company’s investigational gene therapy for the treatment of patients with moderate and severe…

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…

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest.    Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…

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…

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Sigilon Therapeutics’ candidate cell therapy, called SIG-001, for hemophilia A. Orphan drug status aims to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA application…