Showing 3026 results for "hemophilia"

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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 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…

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…

If approved for hemophilia A, the one-time gene therapy Roctavian (valoctocogene roxaparvovec) would provide substantial cost savings per patient compared with other preventive treatments, specifically Hemlibra. That’s according to a draft evidence report issued by pricing watchdog the Institute for Clinical and Economic Review (ICER). An independent non-profit…

My husband, Jared, and I are currently in the process of looking for preschools for our daughter, Cittie. She’s almost 4, but sometimes she acts and speaks as if she’s much older. Cittie often tells us that she wants to go to school. Although we refuse to rush her…

This morning, I took my 16-year-old son, Caeleb, to physical therapy at Carrie Tingley Hospital in Albuquerque, New Mexico. He continues to experience recurring pains from past internal bleeding episodes in his right knee and ankle. The goal is to find the source of the pain and a…