A single infusion of AMT-061, an investigational gene therapy for hemophilia B, continues to maintain clinically significant increases in clotting factor IX (FIX) up to six months after its administration, as demonstrated in all three patients enrolled in uniQure…
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Inspired to sponsor a national conference for women with hemophilia types A and B, The Hemophilia Foundation of Michigan (HFM) became a game changer last November when it hosted an event called “Being Visible.” Gathering 103 women representing 32 states, the event featured physicians…
Two clinical trials launching in December will use Octapharma USA’s Wilate, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection, in patients with hemophilia A. Hemophilia A is an X-linked hereditary bleeding disorder that results from either reduced or absent levels of…
Pfizer is preparing to launch its hemophilia B gene therapy Beqvez (fidanacogene elaparvovec-dzkt) after securing a U.S. approval for the treatment last month. Beqvez is the second gene therapy to be approved for adults with moderate to severe hemophilia B in the U.S., following the clearance of…
Amarna Therapeutics has secured €5 million (about $5.6 million) in new funding, which it plans to use for developing its lead product, AMA005, an investigational gene therapy that aims to restore blood clotting in people with hemophilia B. The goal is to advance the company’s hemophilia B program…
Problems at work, limited engagement in recreational activities, and less relationship satisfaction contribute to a lower health-related quality of life (HRQoL) and greater pain severity, as reported by adults with hemophilia B, according to a study. Findings also showed that anxiety and depression in caregivers of children with this…
“Use the Press’n Seal to cover his bandages when showering!” That’s the text I sent to the wife of a hospitalized friend, who was preparing to be released after an arduous stay. Hearing of his plight with doctors, nurses, and a second surgery was reminiscent of past hospitalizations with my…
Easter Sunday was wonderful. The pipe organ at our church hadn’t worked in four years, but thanks to generous donors, it was finally restored. We revealed it on Easter with a brass quintet and choir, and the music was truly beautiful. As a classically trained oboist, I am deeply…
A conversation Last night I dreamed that I spoke with my father. I asked him how he knew he had a bleed. Did his knee bubble like a soda? Was it tight, but not painful? I yearned for his input, but my alarm sounded before he was able…