The European Medicines Agency (EMA) has accepted BioMarin Pharmaceutical’s request for accelerated assessment of Roctavian, the company’s investigational gene therapy for severe hemophilia A, for a second time. Last year, BioMarin’s regulatory applications for Roctavian (valoctocogene roxaparvovec) got rejected both in Europe and in the…

Lately, my husband, Jared, and I have been working on home improvement goals. Since the pandemic lockdowns began, couples all over the Philippines have been boasting of their improved homes. With many employees now working from home, several people have both the time and the initiative to renovate and…

The first participant has been dosed in a Phase 1/2 clinical trial evaluating marzeptacog alfa activated (MarzAA) to treat bleeding episodes in patients with factor VII (FVII) deficiency, Glanzmann thrombasthenia, and hemophilia A with inhibitors receiving treatment with…

I learned how to be a caregiver from my mother. Ruby Jane loved my children with complete devotion. While I made her a grandmother, she made me the man I am today. I observed her care for not only family but humanity. She spent years serving as the…

Sometimes a person must learn a lesson the hard way before making changes in their life. My youngest son, Caeleb, is 15, and he is discovering some truths about his body. Due to extensive damage in his right knee and ankle from hemophilia, simple things such as running and…

My husband, Jared, and I are fond of advocacy work. Our advocacy for people with chronic and mental illnesses inspires many of the things we do, including the way we run our small business. Our online jewelry store is our bread and butter, but it also is a way…

Hemlibra (emicizumab-kxwh) has been a wonder medicine for many hemophilia patients, including my youngest son. Among the benefits that he receives is a respite from internal bleeds. He now infuses every other week instead of every day. We no longer focus on numbers regarding inhibitor levels, but pray…

The first participant has been dosed in a Phase 3 clinical trial testing marzeptacog alfa activated (MarzAA), an experimental under-the-skin therapy for hemophilia A and B patients with inhibitors. The study, called Crimson-1 (NCT04489537) and sponsored by the therapy’s developer, Catalyst Biosciences, is currently recruiting patients at sites…

I was recently browsing social media when I randomly clicked on a suggested article titled “34 Things You Need to Give Up to Be Successful.” As an entrepreneur, I get lots of suggested links to articles about success in business and life. I reflected upon what I had just…

CSL Behring has closed an agreement giving it global commercialization and licensing rights to AMT-061, an experimental gene therapy for hemophilia B now being tested in a Phase 3 clinical trial. The therapy’s developer, uniQure, will receive an upfront cash payment of $450 million from CSL…