BioMarin Pharmaceuticals is planning to submit marketing authorization applications to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) seeking approval of valoctocogene roxaparvovec, its investigational gene therapy for the treatment of adults with hemophilia A, before the end of the…

Iron deficiency has always been a part of my life as a woman with the bleeding disorders hemophilia B and von Willebrand disease. Iron is essential for your body to function, I have learned. One cause of iron deficiency is bleeding. When I am anemic, or my…

Last week, my wife, Cazandra, my youngest son, Caeleb, and I attended the annual meeting of the Hemophilia Federation of America, held in San Antonio. The grassroots, national organization promotes the health and well-being of those who manage chronic bleeding disorders. While attending the conference, we engaged with…

uniQure N.V., a leading pharmaceutical specialized in human gene therapy, has presented additional data from its clinical trial in hemophilia B patients at the 21st Congress of the European Hematology Association (EHA),  June 9-12, in Copenhagen. The Phase 1/2 clinical trial of AMT-060 titled “Trial of…

Bayer has awarded a combined $2 million in hemophilia research and patient-care grants to 16 scientists and doctors from eight countries. Four of the winners are from the United States. Three represent the Children’s Hospital of Philadelphia and one the Uniformed Services University of the Health Sciences in…

Japan’s Ministry of Health, Labor and Welfare has approved CSL Behring‘s Idelvion (albutrepenonacog alfa) for the prevention of bleeding tendency in patients with hemophilia B. Hemophilia B, a bleeding condition that mainly affects males, is characterized by a deficient or defective factor IX protein, and is treated with intravenous infusion of factor IX.

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to efanesoctocog alfa, an experimental replacement therapy for hemophilia A designed for once-weekly dosing. This designation is given by the FDA to help speed the development and review of treatments for serious or life-threatening conditions. Specifically,…

I am convinced that many of us live for “aha” moments. I’m referring to those times when an unexpected realization occurs that either confirms or changes the course of one’s life. These moments are often welcome guests, but sometimes they rage like a fierce hurricane, and the best we…

Bleeding disorders affect everyone, male or female, no matter how old you are or where you are in the world. If left untreated, bleeding disorders can lead to serious consequences, and may even cause death. Bleeding disorder patients in developing countries do not have any support from their governments in regards to necessary care. In fact, they may not even get help when it comes to being diagnosed. Unfortunately, these conditions often affect children, who needlessly suffer through all the awful symptoms of a bleeding disorder,…

The U.S. Food and Drug Administration (FDA) has approved Shire’s Adynovate for the treatment of pediatric patients under 12 years of age with hemophilia A. Adynovate also was approved for use by both adults and children undergoing surgical procedures, to control bleeding episodes. Adynovate is a recombinant Factor…