As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…

As an expectant mother, I dreamed about my son’s future. I imagined the sound of his voice, the daily rituals we would create together, and the way I’d simply hold and love him with every part of my being. When my first son, Julian, came into the world, my…

My husband, Jared, has fond memories of his school days when he was younger. He went to an all-boys combined elementary school and high school, where roughhousing and horsing around with friends were common. He and his classmates sometimes were crude and rowdy, he says, but they were all very…

My youngest son, Caeleb, became best friends with a girl named Taylor in sixth grade. Then, our family moved away to Texas, but returned to New Mexico two years later. Caeleb and Taylor now live an hour apart. Despite it all, they have remained close. It is remarkable for a…

For people with hemophilia B, switching to Rixubis (nonacog gamma) as a factor IX (FIX) replacement therapy was not associated with increased bleeding rates, dose changes, adverse blood clotting events, or the development of inhibitors among patients with moderate to severe forms of the disease,…

I have a number of friends working as financial advisors. From time to time, they offer me life insurance policies, stock market deals, and other similar investments. As a young mom and businesswoman, I am right within their target market. I take interest in all sorts of financial tools…

A research study is asking people with hemophilia and other inherited bleeding disorders living in the U.K., U.S., or Germany to take part in a survey evaluating how the COVID-19 pandemic is affecting the mental health of those with these diseases. The four-part survey, which can be…

I faced my old friend, the medical supplies boxes, still not believing the task ahead of me. I almost spoke to the six boxes stacked on top of one another. “I am sorry,” I wanted to say, “but we do not need you any longer. We must move on.” Each…

March is Bleeding Disorders Awareness Month. It is a time when organizations such as the National Hemophilia Foundation and the Hemophilia Federation of America encourage the community to share their stories about life with a bleeding disorder. Many use social media to spread awareness by posting…

Enzyre is partnering with Takeda with a goal of developing at-home assays to help people with monitoring congenital bleeding disorders, including hemophilia, and to diagnose these disorders. This partnership builds on an existing, 2019 research collaboration between these…