I recently had the pleasure of watching the “Barbie” movie with my husband and daughter. The experience not only provided us with enjoyable family time, but also left a lasting impact on me. Beyond the vibrant visual effects, comedic gags, and engaging storyline, the movie delivered a profoundly empowering…

The U.K.’s National Institute for Health and Care Excellence (NICE) has issued draft guidance recommending that England’s National Health Service (NHS) should not cover the gene therapy Hemgenix (etranacogene dezaparvovec) for adults with hemophilia B. The recommendation is based on uncertainty surrounding the long-term efficacy of the…

Several of my recent columns centered on my frustration with hemophilia entering my sons’ lives at the most unexpected times. I wrote about some extraordinary moments we shared that, in the twinkling of an eye, were interrupted by an unforeseen health complication. A sense of failure would…

Roctavian (valoctocogene roxaparvovec-rvox), the first gene therapy approved for adults with severe hemophilia A, is expected to be widely available in the U.S. this month, according to its developer, BioMarin. BioMarin also is working with authorities in Germany, Italy, and France to secure market access to…

The piercing bug has bitten me again! After watching TikTok videos of teens and young adults with unconventional piercings, I decided to get a few more myself: a nose (septum) piercing, a center labret piercing, and a couple more ear piercings. My inner child is elated! My…

Nearly a year has passed since Hurricane Ian wreaked havoc on southwest Florida, leaving my hometown of Punta Gorda and its neighboring communities in disrepair. As recovery efforts continue, the renovation of my condo has been stalled due to a failure by our association to reach an agreement with…

Roche is initiating a Phase 3 clinical trial to test the experimental gene therapy SPK-8011 in people with hemophilia A. The company announced the move in its 2023 half-year results. At the same time, however, Roche is discontinuing the development of another investigational hemophilia A gene therapy…

Last week, I stumbled upon the Tour de France while aimlessly flipping through TV channels. This annual professional bicycle race is highly esteemed and considered one of the most difficult events in the sport. Watching it was thrilling. The competition was captivating, and the incredible athleticism of the…

Orsini Specialty Pharmacy has been selected to distribute the new gene therapy Roctavian (valoctocogene roxaparvovec-rvox) for use by individuals with hemophilia A, the company announced in a press release. The one-time treatment from BioMarin Pharmaceutical late last month became the first gene therapy ever approved by…

Dosing has begun in a Phase 2b clinical trial evaluating the safety and efficacy of SerpinPC, Centessa Pharmaceuticals’ investigational therapy for hemophilia. The open-label study, called PRESent-2 (NCT05789524), is part of the company’s registrational program for hemophilia B, which includes multiple clinical trials aiming to…