Last week, my husband, Jared, and I booked a visit to our trusted dentist. We hadn’t had our regular teeth cleaning for over a year because of pandemic restrictions. Alarmed by the amount of tartar on our teeth and frequent toothaches, we thought we’d better go in for dental…

Nearly two-thirds of children with severe hemophilia A given once-weekly Altuviiio (efanesoctocog alfa) in a Phase 3 clinical trial were completely free of bleeds over about one year of treatment. That’s according to new findings from the recently-completed XTEND-Kids study (NCT04759131), which were presented as late-breaking…

Several years ago, I spoke at a great bleeding disorders conference in California. Around 300 people attended, and we all had wonderful interactions. The attendees engaged with one another and found hope in their shared experiences. I left the conference energized and proud to be part of such a…

Dimension Therapeutics may soon become a wholly owned subsidiary of Regenxbio, following the terms of an agreement between the two companies. The merger agreement, if finalized, will add Dimension’s investigational drug candidates being developed to Regenxbio’s pipeline, including DTX201 for the treatment of hemophilia A. DTX201 was being developed…

It’s been almost 30 years since I first discovered the joy of pregnancy. I can still recall my excitement as I left the doctor’s office, clutching the positive test in my hand. I was eager to surprise my husband, Joe, who was teaching high school choir. I called his…

One ordinary evening, while a dear cousin and I relaxed in my living room, we delved into a profound conversation about purpose. We pondered why some people who struggle with mental health would cling to altruistic pursuits despite financial challenges they may have, especially since those challenges could create more…

Second in a series. Read part one. In part one of my interview with Chip Hailey, a 75-year-old from Tennessee, we explored what led him to pursue gene therapy for hemophilia B, his lifelong journey with the condition, his hopes for a new chapter, and the road to…

New preclinical data on one of Catalyst Biosciences’ lead candidate therapies — marzeptacog alfa activated (MarzAA), an engineered clotting Factor VIIa (FVIIa) given as an under-the-skin injection — support its potential to treat acute bleeds in people with hemophilia and its ability to be used safely in combination with Hemlibra…

Two new patients in the U.S. have received Hemgenix (etranacogene dezaparvovec), the first gene therapy to be approved for hemophilia B, per new reports. The therapy was first administered last year in May to a patient at UC San Diego Health, per a University of California, San Diego, news…

A single dose of the experimental gene therapy FLT180a can safely maintain the activity levels of factor IX (FIX) within a normal range for nearly three years, effectively preventing bleeds and the need for replacement therapies in patients with hemophilia B, according to updated data from a Phase 1/2…