I recently picked up a fascinating book, “The Courage to Be Disliked,” by Ichiro Kishimi and Fumitake Koga. The book dives into the philosophy of Alfred Adler, a pioneering figure in individual psychology. Adler’s ideas stand in contrast to the older, trauma-focused theories of…

I’ve always had a hard time handling disappointment. I grew up labeled a high achiever and was accustomed to meeting high expectations. I feared disappointing other people, or feeling that my best efforts just weren’t enough. As a result, I became sensitive to disappointment, rejection, and failure — a fearsome…

The U.S. Food and Drug Administration (FDA) has accepted Bioverativ’s investigational new drug application (IND) for BIVV001, an investigational Factor VIII therapy designed to extend protection from bleeds in hemophilia A patients. The FDA will now review the IND application for safety. The Waltham, Massachusetts-based company plans to initiate a Phase…

Last week, my husband, Jared, experienced a bleed above his knee joint. We were out on a day trip when he noticed the first symptoms. He felt a tightness in his knee, which he initially attributed to climbing several flights of stairs. It wasn’t painful for him, but as…

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to BioMarin Pharmaceutical’s investigative therapy, valoctocogene roxaparvovec to treat patients with hemophilia A. This new status is likely to speed up the development and regulatory review of valoctocogene roxaparvovec, which has already received an Orphan Drug Designation from both…

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…

From early on, we find success by developing a strong work ethic. Some of us come by it naturally, while others learn the value of working hard and doing the best job they can do. I realized that discipline brought great rewards, both musically and academically. When I failed to…

Valoctocogene roxaparvovec, BioMarin Pharmaceutical‘s investigational gene therapy, results in a more than 90% drop in annual bleeding rates and the use of clotting factor VIII (FVIII) in men with severe hemophilia A, updated data from a Phase 1/2 clinical trial show. It also precluded the need for…

The added expenses of a bleeding disorder — beyond medical costs — are rarely discussed in our community. Yes, I’m talking about the dreaded M-word, which can evoke many negative emotions. Money affects every part of our lives, and for some of us, our relationship with it results in feelings…

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…