A dreary day is unusual in the desert of New Mexico, where we live. When it happens, the sky opens, releasing a much-needed downpour of liquid gold. The dry ground is soaking up this scarce commodity. It’s a bad hair day for many who aren’t used to double-digit humidity, and…
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BioMarin Pharmaceutical has resubmitted an application to the U.S. Food and Drug Administration (FDA) seeking approval of Roctavian (valoctocogene roxaparvovec), potentially the first gene therapy for adults with severe hemophilia A. The FDA delayed the company’s initial approval request in 2019, with the agency requiring two more…
My son Caeleb participated in a marching band competition this past Saturday. He plays the keyboard in the pit so that he doesn’t have to move around on the field, which would cause intense pain. Many years of internal bleeding left no cartilage in my son’s damaged right…
I like to watch jugglers. From balls and plates, to bowling pins and swords, performers toss items into the air one after another, never letting them hit the floor. Their hand-eye coordination is tremendous, as well as their ability to move, twirl, and even ride a unicycle while keeping the…
I discovered that I have a talent unlike any other. I’m a world champion at throwing pity parties. When times get tough and I feel lost, all I must do is put a cone-shaped hat on my head and recognize my misfortune. My glum permeates the air as I raise…
I have a version of Pandora’s box. Fortunately, the box doesn’t see the light of day. I keep it on the top shelf of my closet, hidden behind extra pillows that are stored away for months on end. The box contains years of pain, grief, sorrow, and buckets of tears.
Men with moderate-to-severe hemophilia A are again being enrolled in the Phase 3 AFFINE trial, which is testing the safety and effectiveness of SB-525 (giroctocogene fitelparvovec), an experimental gene therapy being developed by Pfizer and Sangamo Therapeutics. Pfizer, the study’s sponsor, had placed a voluntary pause…
My husband, Jared, and I are just starting to figure out family life. As novices to the world of parenting and homemaking, we’re navigating our child’s formative years and learning to maintain a household through trial and error. Living by ourselves has been an especially helpful experience. Choosing not…
The end of 2022 is rapidly approaching, and I’ve made nary a dent in my fitness and diet goals for this year. Oh, sure, I have a ton of excuses — like my foot that swells up when I walk more than a mile — but there really…
Treatment with marstacimab (PF-06741086), an experimental antibody-based therapy developed by Pfizer, reduced the number of bleeds in patients with severe hemophilia who took part in a Phase 1b/2 clinical study. This held true regardless of whether patients had hemophilia A or hemophilia B, or had inhibitors…