The U.S. Food and Drug Administration has approved new vial strengths for Octopharma’s hemophilia A therapy Nuwiq, which means patients will need fewer vials per treatment session. Octapharma USA said single-dose strengths of 2,500, 3,000 and 4,000 International Units will be available in the United States in September. The vials are already…
Mononine, or human coagulation factor IX, is a factor IX concentrate indicated for the prevention and control of bleeding in hemophilia B. The U.S. Food and Drug Administration (FDA) approved its use in 1992, making it the first highly purified factor IX on the U.S. market.
The first patient in a Phase 1/2 clinical trial (“the Alta trial”) designed to assess safety and tolerability of SB-525 gene therapy has received treatment. Sangamo Therapeutics, in a collaboration with Pfizer, is developing  the SB-525 gene therapy to correct the Factor VIII (FVIII) defect in hemophilia A…
The U.S. Food and Drug Administration (FDA) has granted priority review to the therapy emicizumab for patients with hemophilia A with factor VIII inhibitors, Genetech, the drug’s developer, announced. The FDA also announced the acceptance of Genetech’s Biologics License Application (BLA) for emicizumab prophylaxis (preventive) therapy in adults, adolescents and children…
Last updated Dec. 10, 2024, by Susie Strachan Hemophilia has affected people from all walks of life, including royalty, athletes, actors, and advocates. It’s a rare genetic bleeding disorder that mostly affects males, while females are more likely to be carriers. Learn about some of the notable names…
Recent advances in gene therapy approaches to treat hemophilia B were recently announced by uniQure. The company achieved “substantial progress in advancing our lead program in hemophilia B,” Matthew Kapusta, CEO of uniQure, said in a press release. The company reacquired the development and commercial rights to…
https://www.youtube.com/watch?v=pTIVORH7anc This video from Wochit News is all about a new treatment for severe hemophilia A. The Children’s Hospital of Los Angeles, California, has developed a breakthrough treatment that can reduce the bleeding episodes in people living with hemophilia A by up to 90 percent. MORE: Emicizumab shows…
Three patients who received SPK-8011 — a gene therapy developed by Spark Therapeutics — for hemophilia A show increased production of clotting factor VIII without any safety issues reported, says the company. The patients are the first to receive the treatment as part of a Phase 1/2 clinical trial, with…
Discovering your child has hemophilia can be devastating. While you will certainly need to make some lifestyle changes moving forward, with the correct treatment and preventative measures, there’s no reason why your child can’t live a happy and healthy life. To help you come to terms with life with hemophilia and…
In order to determine if a person has hemophilia, they will have to undergo various tests to see if their blood is clotting as it should be. There are four different types of blood screening tests that are used to diagnose hemophilia: Complete Blood Count A complete blood…
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