All five patients in an early-stage trial given the one-time treatment, giroctocogene fitelparvovec, did not have spontaneous bleeding episodes or require infusions of the blood-clotting protein they otherwise lack, the companies said. Pfizer and Sangamo's therapy is one of several treatments being developed for hemophilia A, as an alternative to the current practice that requires regular infusions to replace the missing protein, factor VIII. On Wednesday, a rival gene therapy by BioMarin Pharmaceutical Inc was found to reduce bleeding episodes by over 90% in patients treated four years ago....
Akili Interactive’s video game “EndeavorRX” is a digital therapeutic treatment designed for children with ADHD. After 7 years of clinical trials, studying 600 children, the FDA cleared the video game as medicinal therapy....
