I couldn’t believe Caeleb was about to get his first dose of Hemlibra (emicizumab-kxwh). I have been following several people whose children participated in the Hemlibra study (NCT02622321) before the Food and Drug Administration approved the therapy, and I saw how it changed their lives. After…

Hemophilia A patients who do not refrigerate their bleeding prevention therapy are more satisfied with it and report fewer restrictions on their daily activities, a study finds. Researchers also discovered that most of the patients were unaware that they could keep their recombinant factor VIII clotting therapy at room temperature.

When my sons were born and diagnosed with hemophilia, we lived in Houston and had access to the Gulf States Hemophilia and Thrombophilia Center. I had access to treatment and never considered that I’d be kept from it. When my youngest, Caeleb, was only 7 months old, my…

In honor of Bleeding Disorder Awareness Month, celebrated in March, CVS Health is donating more than $260,000 to national and local hemophilia and bleeding disorder programs across the United States. Although there’s no exact number of how many people are now living…

There are moments that forever alter lives. Sometimes we celebrate educational goals, while others encounter the excitement of new love. Other times leave us breathless as we attempt to rise again from a catastrophic event, unsure whether we will ever recover. Whatever we experience, our worlds change and none of…

  I have a few rituals on Sunday evenings. I check to make sure clothes are washed and ready for the week, I set the menu so that we have a plan for dinner throughout the week, and I fill the infusing bucket and bring it to the…

A delivery method using nanotubes with a spear-like shape may open new avenues for safer, faster, and more cost-effective gene therapies for several genetic diseases, including hemophilia, according to a study. The study, “Precision-Guided Nanospears for Targeted and High-Throughput Intracellular Gene Delivery,” was recently published in the…

  I had the amazing privilege of speaking to the women of the Gateway and Midwest hemophilia associations at their annual retreat for women with bleeding disorders. There is just something about folks in the Midwest. They are kindhearted, compassionate, and tons of fun. Every time I…

BioMarin Pharmaceutical has won an award for getting its new gene therapy facility in Novato, California, operational ahead of schedule. The International Society for Pharmaceutical Engineering (ISPE) award was for project execution. It was part of the society’s 2018 Facility of the Year honors. BioMarin took only a year from the time…

Hemlibra (emicizumab) is an effective and less costly therapy for hemophilia A patients who have substances in their blood that prevent clotting factor therapies from working as well as they should, according to a therapy rating organization. The Institute for Clinical and Economic Review (ICER) updated an initial report on Hemlibra…