People are required to earn a certification or license for numerous activities. To drive a vehicle, a person must secure a learner’s permit or driver’s license. To become a lifeguard, a person must go through training to earn a certification. Even working a part-time job in the food industry…

“Beep, beep.” I leap out of bed at 10 a.m. on a Saturday. My plans for the day include geocaching, a wonderfully nerdy hobby in which I hike through forests and urban areas to find hidden boxes. The GPS on my phone leads me…

Bayer’s Jivi safely and effectively prevents bleeds during and after minor surgery in children, adolescents, and adults with severe hemophilia A, according to interim data from the Phase 2/3 PROTECT VIII and the Phase 3 PROTECT VIII Kids clinical trials. Most of the included patients were on…

Preventive, or prophylactic, treatment with Hemlibra (emicizumab) safely and effectively managed bleeding in adults and children with severe hemophilia A, a real-world study has found. The study, “Emicizumab prophylaxis: Prospective longitudinal real‐world follow‐up and monitoring,” was published in the journal Haemophilia. Originally developed by Roche’s subsidiary…

Screening for clinical trials in gene therapy can exclude the vast majority — 92% — of severe hemophilia patients, for reasons too often due to potentially modifiable factors such as socioeconomic status and location, a study of patients at a treatment center in Belgium reported. Patients’ unwillingness to…

The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on uniQure’s gene therapy program for hemophilia B, which includes AMT-061 (etranacogene dezaparvovec), the company announced. According to uniQure, the FDA found it was “very unlikely” that AMT-061 had contributed to a case of liver cancer in a patient who…

My husband, Jared, has a complex medical situation: He has severe hemophilia B and a seizure disorder, which both greatly affect his daily life. He recently started online consultations with a psychologist, a psychiatrist, and a neurologist to help him work through his seizure disorder, which was caused by a…

Healthcare costs can be 25 times higher for hemophilia B patients than for individuals who do not have a bleeding disorder, according to a recent study. These findings reflect both direct costs of hemophilia treatment and a greater need for other healthcare resources, researchers noted. The study, “…

Scientists found the immune signaling protein BAFF can promote the formation of neutralizing antibodies, or inhibitors, against blood clotting factor VIII (FVIII), which can lower the effectiveness of FVIII replacement therapies used to treat people with hemophilia A.  They also discovered that hemophilia A…