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The U.S. Food and Drug Administration (FDA) approved Sevenfact (coagulation factor VIIa [recombinant]-jncw) for the treatment and control of spontaneous bleeding episodes in people, ages 12 and older, with hemophilia A or B with inhibitors. Hemophilia, a genetic disorder, affects the body’s ability to make blood…

A Phase 3 clinical trial investigating gene therapy candidate AMT-061 (etranacogene dezaparvovec) has reached its target of dosing 50 participants with moderate or severe hemophilia B. In fact, according to AMT-061 developer uniQure, 54 patients have been given the one-time dose of AMT-061 in the HOPE-B…

Endothelial cells — those that line the inside of blood vessels — derived from stem cells of people with hemophilia A and modified to produce functional factor VIII were able to ease blood loss in a mouse model of the disease, a study reported. These findings support the potential of induced…

Some $80.3 million in financing will support the first clinical trial of Sigilon Therapeutics’ new hemophilia A cell therapy candidate, SIG-001, the company said. The trial is expected to begin by June. Last August, the U.S. Food and Drug Administration (FDA) granted the potential cell therapy…

It’s a struggle to balance life with two disorders. I need to take daily medications for epilepsy while also self-infusing to treat internal bleeds. I recently suffered a horrible epileptic episode. My wife, Cza, and I were walking with our baby, Cittie, when I felt a strong aura warn…

COVID-19, short for coronavirus disease 2019, is an infection caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), a newly identified and highly contagious pathogen not previously seen in humans. Though it belongs to the same category of viruses as SARS and influenza…