The summer months bring relentless heat. In the desert of New Mexico, where we live, the thermometer often hits triple digits. While some people love the heat, others tolerate it, seeking shelter in the shade of a tree or in an air-conditioned area. Staying cool isn’t just a…
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BeneFIX (nonacog alfa) is a recombinant, or man-made, clotting factor therapy approved to prevent and treat bleeding episodes, including those occurring during surgery, in adults and children with hemophilia B.
The Spanish Interministerial Commission on the Pricing of Medicines, known as CIPM, has issued a positive recommendation for national reimbursement of Hemgenix (etranacogene dezaparvovec) for eligible people with hemophilia B, according to CSL Behring, the company that markets the gene therapy. The recommendation means that patients in Spain should…
My mighty warrior Caeleb came home from school last week with a question. “Mom,” he asked, “my knee is really messed up, isn’t it?” I couldn’t see an injury, and so I asked him what he meant by “messed up.” “I can’t run really fast like everyone else in gym class,”…
Through my journey of being an advocate for two sons with hemophilia, I’ve continued to learn life lessons that enrich my soul and make me a better person. While it’s true that raising Julian and Caeleb involved the heavy burden of managing their bleeding disorders, I’ve discovered…
A gene therapy approach acquired by uniQure was recently covered in two new patents, one issued in the United States and one in Canada, broadly covering ways of treating bleeding disorders that include hemophilia B, the company announced in a press release. The therapy has a nucleic acid encoding the…
The U.S. Food and Drug Administration (FDA) has granted uniQure’s AMT-060, an investigational gene therapy, breakthrough therapy designation for patients with severe hemophilia B. The regulatory agency based its decision on data from the ongoing, dose-ranging Phase 1/2 clinical trial (NCT02396342) evaluating AMT-060 for hemophilia B. The study’s estimated completion date…
The other day, I asked my husband, Jared, if he’d ever consider leading a harm reduction seminar for the teens in his hemophilia organization. His immediate response was, “Of course — but would the parents be ready for that conversation?” That question hit me. As…
The widespread adoption of telehealth during the COVID-19 pandemic has improved access to specialty care for people with hemophilia, made it more convenient, and increased patient engagement, a study based on surveys reported. Healthcare providers at an Irish care center also…