Showing 2917 results for "hemophilia"

Phase 3 Trial of SB-525, Gene Therapy for Hem A, Again Enrolling

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…

Hem A Gene Therapy Roctavian More Cost-effective Than Hemlibra

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…

Walking Down a Hospital Hallway Brings Back Memories

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…