I vividly remember the playground at Our Lady of Fatima Catholic School in Galena Park, Texas. The monkey bar dome was the place for the risk-takers, who climbed to the top and faced the ground. I preferred the swings, which catapulted the brave ones as they flew off in a…
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Note: This story was updated March 28, 2024, to clarify the gene therapy delivers the mRNA of the Cas9 enzyme. Intellia Therapeutics is ending its collaborative agreement with Regeneron, which had the two companies working together in developing hemophilia B gene-editing therapies, according to a securities and exchange…
After I had spent a few days lobbying legislators in Washington, D.C., my youngest son, Caeleb, joined me for some much-needed time together. I met him at the airport to start our madcap adventures. He was in a wheelchair due to chronic pain from numerous spontaneous bleeding…
AlphaNine SD (human coagulation factor IX) is a highly purified, plasma-derived clotting factor IX (FIX) concentrate that's approved to prevent and control bleeding in people with hemophilia B.
Creating personalized hemophilia B care is an important part of hemophilia B management.Your healthcare team can set up a treatment plan based on your specific needs and the severity of your symptoms.
Nearly all medical directors at hemophilia treatment centers in the U.S. said they would consider starting preventive Hemlibra (emicizumab-KXWH) in untreated infants with hemophilia A, ages 12 months and younger, a survey study reports. Most respondents also said they’d transition from FVIII replacement therapies, used on-demand to treat…
ReciBioPharm and GeneVentiv Therapeutics are teaming up to advance the development of GENV-HEM, a gene therapy for all hemophilia patients, including those who have developed inhibitors against their missing clotting factors. Developing a gene therapy requires efficient, flexible, and fast manufacturing, and each step…
Rixubis (nonacog gamma) is a replacement therapy approved to prevent and treat bleeds, including those occurring during surgery, in adults and children with hemophilia B.
Last Thursday, I visited Washington, D.C., to join my fellow blood brothers and sisters in lobbying legislators regarding issues that directly affect the bleeding disorders community. I must admit that I was a little nervous. Several times, I wondered, “What in the world am I doing here? I’m only…