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…

I am still waiting to hear from our hemophilia treatment center about whether our insurance will cover Hemlibra (emicizumab-kxwh) for Caeleb. Honestly, I’ve put it out of my mind for a bit because I know it is not a “done deal” until I have the product in my…

Precision BioLogic has developed what it says is a better way of detecting levels of substances in hemophilia A patients’ blood that prevent clotting factor therapies from working as well as they should. It collaborated with Roche and Genentech in creating a kit it says can detect levels of the…

Last week, my husband and I took our mighty warrior Caeleb to the hemophilia treatment center so that we could touch base with his hematologist. Caeleb recently had labs drawn, and it was time to sit for a meeting about his treatment. We were called to the back to…

The National Hemophilia Foundation (NHF) kicked off its 70th anniversary with an advocacy effort on Capitol Hill in Washington, D.C. At the same time, the NHF launched its 2018 Red Tie Campaign  to raise awareness about Bleeding Disorders Awareness Month and reinforces its campaign to oppose the U.S. Congress’ efforts to terminate…

Hemophilia in children is diagnosed at an average age of 9 months, and most children are diagnosed by their second birthday. If your pediatrician suspects your child has hemophilia, you may be concerned about their first appointment with the hematologist. To ease your worries, we’ve put together some tips to help you through…

myPKFiT for Advate, a free web-based software designed to personalize and assess preventive dosing regimens for some hemophilia A patients treated with Advate, is now available in the U.S. MyPKFiT for Advate is the first and only pharmacokinetic (PK) dosing software approved by the U.S. Food and Drug Administration (FDA)…