In an effort to cut costs and boost profitability, Biomarin Pharmaceutical is limiting commercial development of hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec-rvox) to three countries: the U.S., Germany, and Italy. The company has struggled to turn a profit from the gene therapy, which was approved in…

I met Jennifer Hastie last year at the Hemophilia Federation of America’s Mild Matters Summit in Tulsa, Oklahoma. Along with two of her three daughters, Jennifer has von Willebrand disease (VWD) type 2B, a rare variant. We share more than just a name: Jennifer is also 58…

As a pastor, my Sunday mornings prove very busy. I lead morning worship at 9 a.m., teach a Sunday school class at 10:15, and finish my morning with another service at 11. Most Sunday afternoons I can be found collapsed in my comfortable recliner and dozing. I’ll be exhausted from…

As a woman of faith, I look for God in various places. I experience God in a beautiful sunset, a phone call from a dear friend, or even in the middle of a struggle. Walking a labyrinth is one of my favorite places to connect with God. A labyrinth…

Game shows have experienced a resurgence in recent years, captivating audiences with their blend of entertainment, competition, and nostalgia. I’ve always liked game shows, especially those involving trivia. In “Who Wants to Be a Millionaire?,” contestants answer multiple-choice questions; if they’re correct, they advance to the next level, with…

Nearly two-thirds of children with severe hemophilia A on preventive, or prophylactic, treatment with once-weekly Altuviiio (efanesoctocog alfa) in the yearlong XTEND-Kids study were free of bleeding episodes needing treatment. More than 80% in this Phase 3 clinical trial also were free of spontaneous bleedings and joint bleeds needing…

I recently stumbled upon a thought-provoking post by “Typical Pinoy Crap” (TPC), a well-known Filipino digital creator who shares insightful views about social issues. The post commented on a line from a song by the all-girl Filipino pop group BINI, titled “Karera” (“Race”). The line is…

Cazandra and Caeleb, my wife and youngest son, rode with me several years ago to see a movie in Albuquerque, New Mexico, near our home. Caz and I planned the whole day with one goal: to have fun. After the movie, we’d have lunch and then go to a…

I recently received an intriguing piece of jewelry in the mail. It’s a prototype of a design I hope to sell someday. But I loved it so much that I wanted one for myself. The design is rather goth-themed, featuring a coffin-cut black onyx gem in the center flanked…

In a reversal of a previous recommendation, the U.K.’s National Institute for Health and Care Excellence (NICE) has recommended that the National Health Service (NHS), England’s publicly funded health insurance program, should offer reimbursement for Hemgenix (etranacogene dezaparvovec) to eligible adults with hemophilia B. Reimbursement would come through…