The U.S. Food and Drug Administration (FDA) has granted orphan drug status to SerpinPC for treating hemophilia B. Orphan drug status is awarded to therapeutics intended to treat rare conditions, defined as those affecting fewer than 200,000 people in the U.S. The designation provides financial incentives to support clinical…
Search results for:
Read about Esperoct, an extended half-life infusion treatment approved to prevent or stop bleeding in children and adults with hemophilia A.
Social Clips
A Brief History of Hemophilia Treatment
The first recorded mention of hemophilia came in the second century when a collection of ancient Jewish writings noted that baby boys did not need to be circumcised if two of their older brothers had bled to death following the procedure. Later in the 10th century, an Arabian doctor described cases of…
As gene therapy is relatively new treatment option for hemophilia B, it's normal to have some questions about it. Here are answers to some commonly asked questions about gene therapy for hemophilia B.
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,…
Hemlibra (emicizumab-KXWH) is an antibody-based therapy used to prevent or reduce bleeds in hemophilia A patients, with or without factor VIII (FVIII) inhibitors. It is given as an under-the-skin, or subcutaneous, injection.
The spleen’s marginal zone (MZ) B-cells respond to blood coagulation factor VIII (FVIII) and show potential to become the target of future therapies to reduce FVIII inhibitors in hemophilia A, according to new research in mice. The study, “Marginal zone B cells are critical to factor VIII inhibitor…
Ease of use, fear of the unknown, and healthcare providers’ advice are all major factors that play a role in whether people with hemophilia want to switch to new treatment options, a small study suggests. The study, “Patient Perspectives on Novel Treatments in Haemophilia: A Qualitative Study,”…
Rare Disease Day, observed this year on Feb. 29, serves as a poignant reminder of the challenges facing those who live with uncommon medical conditions. According to the event’s website, a rare disease is defined as one that affects fewer than 1 in 2,000 individuals. Among these conditions…
Qfitlia (fitusiran) is an RNA-based therapy that is approved to prevent or reduce the frequency of bleeds in patients with hemophilia A or B, with or without inhibitors. It is given via under-the-skin, or subcutaneous, injections that can be self-administered.