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…

I was over the moon when my first son, Julian, was born in 1996. My baby was here, and life would never be the same. When Julian wouldn’t stop bleeding after his circumcision, the pediatrician was visibly shaken. Seeing a doctor with furrowed eyebrows is disconcerting. The next day,…

In a recent conversation with my husband, Jared, he dropped a bombshell. If he were given the chance to be rid of hemophilia entirely for the rest of his life, he said he’d hesitate to take it. He’d actually miss his current life with severe hemophilia B. My…

Note: This column shares a woman’s self-described experience administering herself factor despite a doctor’s advice. Consult your doctor before starting or stopping any therapy. For the first time, I attended the Coalition for Hemophilia B’s annual symposium, held late last month in Dallas. It was an enriching experience where…

I never understood why graduating from high school was such a big deal. I grew up in a home of educators, and getting good grades and attending college were expected. Not pursuing higher education was never a consideration. I excelled academically and enjoyed my high school years; they were some…

Gentle electrical stimulation may help liver cells uptake gene therapies, such as those used to treat hemophilia, according to new research by a team of U.S. scientists. In lab studies, the team found that liver cells exposed to a brief electrical field more easily took up a viral carrier,…

My husband pastors the local First United Methodist Church, where I serve as a deacon. When a parent (or two) goes down the pastoral path, their children are along for the ride. Some preachers’ kids love church, and others, not so much. My youngest son, Caeleb, falls…

The U.S. Food and Drug Administration (FDA) has approved the one-time gene therapy fidanacogene elaparvovec for treating certain adults with moderate to severe hemophilia B. The therapy will be sold under the name Beqvez as it is in Canada, where it was approved for a similar indication this…

GeneVentiv Therapeutics has received a $2.5 million grant to conduct further preclinical tests on its one-time infusion gene therapy for hemophilia A and B patients with or without clotting factor inhibitors. The therapy, GENV-HEM, will be tested in a large study with a canine model of hemophilia, following…

The Hemophilia Federation of America recently hosted its annual symposium in Indianapolis. Despite my desire to attend, circumstances didn’t align for me this year. One of the event’s highlights was the debut of digital content agency Believe Limited‘s latest cinematic offering, “On the Shoulders of Giants,” now…