A few weeks ago, my oldest son, Julian, called me from the Blue Ridge Mountains in North Carolina and informed me that he had throbbing pain in one of his teeth. He immediately went to a nearby dentist and got news he didn’t want to hear: The tooth…
One of my favorite movies is “Galaxy Quest,” a comedy that parodies and pays homage to science fiction films and TV. In it, the cast members of a cult series much like “Star Trek” are drawn into an interstellar conflict by real aliens who think the series is…
Prophylactic treatment for hemophilia can help people with the disease avoid unemployment due to disability, a study reports. Hemophilia has a negative effect on a person’s employability. A 2022 global report by the World Federation of Hemophilia indicates about 18% of hemophilia patients see their employment status impacted…
Navigating life with hemophilia in the Philippines presents unique and daunting challenges. The financial burden of treatment is one of the most significant hurdles hemophiliacs face. A 1,000-unit vial of factor IX costs up to 22,000 Philippine pesos (about $377), yet the minimum monthly wage is…
A 39-year-old man from Arizona became the first patient in Nevada to receive Hemgenix (etranacogene dezaparvovec), the first gene therapy to be approved for hemophilia B. The infusion was administered at the Cure 4 The Kids Foundation, under the supervision of Aimee Foord, director of the foundation’s bleeding…
Last updated Aug. 1, 2024, by Marisa Wexler, MS  Fact-checked by Joana Carvalho, PhD What is Novoeight for hemophilia? Novoeight (turoctocog alfa) is a recombinant, or man-made, clotting factor replacement therapy approved to prevent and treat bleeds, including those occurring during surgery, in people with hemophilia A.
SPK-8011, also known as dirloctocogene samoparvovec, is an experimental gene therapy for hemophilia A. Given as a one-time intravenous or into-the-vein, infusion, it aims to reduce the risk of bleeds in patients.
Durveqtix (fidanacogene elaparvovec), a gene therapy for adults with severe and moderately severe hemophilia B, has been granted conditional marketing authorization by regulators in the European Union. The decision by the European Commission follows a positive recommendation on the …
My husband, Jared, and I have been working on some big home renovations lately. Under the supervision of my dad, who is a civil engineer, Jared learned how to do various jobs. He rerouted some water pipes, plugged up some holes, and painted. He also watched as an electrician installed…
Note: This story was updated July 30, 2024, to clarify patients participating in AFFINE had moderately severe to severe disease, and that a secondary study goal involved the mean treated annualized bleeding rate. Giroctocogene fitelparvovec, an investigational gene therapy for adults with hemophilia A, is generally well…
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