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…
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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…
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…
More than 1,300 people who have been affected by acquired immune deficiency syndrome (AIDS) gathered at the National AIDS Memorial in San Francisco on Dec. 1 to commemorate World AIDS Day. During two days of events, family and friends paid tributes to those lost to the disease, while inspiring remembrance…
Caeleb MacDonald shares how hemophilia has shaped his approach to exercise, from understanding physical limits to finding ways to stay active through biking.
Walking into a new hematology visit can feel overwhelming, but preparation and clear communication can help you feel more in control. This guide shares simple ways to advocate for yourself and build a care partnership that truly listens.
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…
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…
Treatment with the extended half-life (EHL) therapies Eloctate (efmoroctocog alfa) and Alprolix (eftrenonacog alfa) was found to safely and effectively control bleeding during major orthopedic surgeries in people with hemophilia A or B, according to real-world data from two Italian centers. The therapies helped patients maintain…
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…