Forty-six. That’s how old I was when I finally accessed the hemophilia care that I’d needed my entire life. I believe the reason it took so long was that I’m a woman. It may seem crazy to those who aren’t familiar with the bleeding disorder. For years, the misconception…

A new study suggests that whole body vibration training increases muscle strength, bone mineral density, and functional capacity in children with hemophilia. The study, “Effect of whole body vibration training on quadriceps strength, bone mineral density, and functional capacity in children with hemophilia: a randomized clinical trial,” was published…

Genetic variations in certain immune-related genes, namely the HLA and IL-10 genes, are linked with an increased risk for developing inhibitors against factor VIII replacement therapies in patients with severe hemophilia A. The study that determined that finding, “Risk stratification integrating genetic data for factor VIII inhibitor development…

Dear 11-year-old me, Life might feel overwhelming at this moment, but remember, you are stronger than you realize. The road ahead will present challenges, but they will shape you into an incredibly resilient person. Your journey with your period has begun, and I know it’s tough. Those…

UniQure has taken major steps this year toward U.S. and European regulatory approval of AMT-060, its gene-therapy treatment for hemophilia B. In January, the U.S. Food and Drug Administration (FDA) gave AMT-060 breakthrough therapy designation for patients with severe hemophilia B. In April the European Medicines Agency (EMA) gave it PRIME designation. The designations…

The European Medicines Agency (EMA) has granted orphan medicinal product status to SB-525, a clinical stage gene therapy candidate under development for the treatment of hemophilia A, according to Sangamo Therapeutics and Pfizer. The announcement coincides with the opening of patient enrollment for a Phase 1/2…

Light Chain Bioscience, a unit of Swiss biotech Novimmune, has completed the discovery phase of its collaborative program with Takeda for developing bispecific antibodies — protective proteins produced by the immune system — that mimic the activity of blood clotting factor VIII as a treatment for…

UniQure has enrolled the first patient in its Phase 3 HOPE-B single-dose trial investigating the effectiveness and safety of AMT-061 in treating people with severe or moderately severe hemophilia B. AMT-061 uses a viral vector to deliver the gene for a mutated clotting factor IX (FIX), which is also known as…

I recently spent an afternoon with a group of parents raising young adults with hemophilia. They were part of a newer generation, people who grew up with a little more information, a little more community support, and slightly more medical options than what my husband, Jared, had access…

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…