A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A — and, in 16 of 18 male patients, those levels were sustained for up to four years, according to results from a Phase 1/2 clinical…

GreenGene F (beroctocog alfa), a lab-made version of coagulation factor VIII, has been approved by regulators in China for the control and prevention, or prophylaxis, of bleeding episodes associated with hemophilia A, the therapy’s developer, GC Pharma,  announced. GreenGene F will be available in China in the first…

Researchers at the Center for Research in Transplantation and Immunology in France have reviewed a paper concerning certain strategies utilized to correct hemophilia B and the importance of gene delivery vehicles in successful therapies for treatment of the condition. The review paper, “Successful correction of hemophilia by CRISPR/Cas9 genome…

I recently visited the small town of Arcadia, Florida, which is not far from my home. Arcadia holds an important place in history for the topic of hemophilia and AIDS. The story of what happened there is a lesson about how ignorance can fuel hatred and knowledge…

Precision BioLogic revealed promising data on a new kit for the measurement of FVIII inhibitors (factor VIII) in patients with hemophilia A. The results were presented at the International Society on Thrombosis and Haemostasis’ Scientific and Standardization Committee (SSC) meeting July 18-21 in Dublin, Ireland. Researchers presented their…

My husband, Jared, and I are two weeks into our self-sufficiency journey. Just last week, our family moved into a rented condo. We spent some time and money to fix up the place and repair damages. So far, Jared’s handyman skills have improved a great deal. He likes that…

It’s a struggle to balance life with two disorders. I need to take daily medications for epilepsy while also self-infusing to treat internal bleeds. I recently suffered a horrible epileptic episode. My wife, Cza, and I were walking with our baby, Cittie, when I felt a strong aura warn…

Hemophilia is a genetic disorder that prevents blood clotting. Most common in men, there are two main types of hemophilia: hemophilia A and B. Below is a curated list of recommended reads for family members, friends and carers of patients with hemophilia, with help from Good Reads and Alibris.

My daughter, Cittie, is now 3 and a half years old. She’s grown so much over the past years, from a tiny and helpless little baby to a more independent preschooler with a mind of her own. She’s gotten good at expressing what she likes and dislikes. She’s also…

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…