Beqvez (fidanacogene elaparvovec-dzkt) was a gene therapy that was approved in the U.S. for the treatment of certain adults with moderate to severe hemophilia B. The one-time therapy, given via an into-the-vein or intravenous infusion, was marketed by Pfizer, who made the decision to halt its development and commercialization.
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While cleaning up recently, I found a physician-authored exercise guide from 2002 for Filipinos with hemophilia. It was fascinating to read, especially knowing that at the time, synthetic factor products were still scarce here in the Philippines. Treatment options were limited, and many people with hemophilia still relied…
The U.K.’s National Institute for Health and Care Excellence (NICE) has recommended Altuvoct (efanesoctocog alfa) be available through the National Health Service (NHS), England’s public healthcare system, as a treatment option to manage and prevent bleeds in people with severe hemophilia A, ages 2 and older. This decision…
Note: This column describes the author’s own experiences with surgical procedures. Not everyone will have the same response to these operations. Consult your doctor about the potential risks and benefits of any procedure you’re considering. As I get older, the thought of needing any kind of surgery scares the…
Second in a series. Read part one. A hemophilia inhibitor is like a firewall in a computer network. Clotting factor replacement therapy is supposed to enter the bloodstream and help form clots to stop bleeding, just as data should pass through a network without interruption. However, an inhibitor acts…
Gene therapy has long been touted as a revolutionary hope for people with hemophilia B, promising a one-time treatment to drastically reduce — or even eliminate — the need for frequent infusions. Yet that hope has hit another roadblock. Pfizer announced last week that it’s halting development of…
Pfizer said it will stop development and commercialization of hemophilia B treatment Beqvez (fidanacogene elaparvovec-dzkt). “Pfizer has made the decision to cease further development and commercialization activities with respect to Beqvez for several reasons, including the limited interest patients and their doctors have demonstrated in hemophilia gene therapies…
Recently, my wife, Cazandra, and I entered an extraordinary time — when our adult sons are enjoying a period of peace with their bleeding disorders. Several months passed, and the only health issues that came into focus were dental surgery for Julian, our oldest, and some minor discomfort for…
First in a series. Every person is unique, shaped by the combination of their upbringing, education, cultural influences, values, attitudes, and behaviors. No two people are exactly alike. Even identical twins, while genetically similar, have differences, including their fingerprints. Medical conditions can also emphasize a person’s individuality. While many may…
The song “The Daring Young Man on the Flying Trapeze,” lyrics by George Leybourne and music by Gaston Lyle, plays loudly in my mind. As a child, I loved the lines “He’d fly through the air with the greatest of ease/ That daring young man on the flying…