Nasdaq1mon
Novo Nordisk A/S: Once-weekly Mim8 is well-tolerated and efficacious in children living with haemophilia A with and without inhibitors
There are different types of haemophilia, which are characterised by the type of clotting factor protein that is defective or missing. Haemophilia A is caused by a missing or defective clotting ...
THE WEEK8mon
A 'transformative' gene therapy for haemophilia B
The NHS can now offer patients with a rare blood disorder a "transformative" new treatment after the UK's medicines watchdog gave its funding the green light. Around 200 people with haemophilia B ...
pharmaphorum2d
Pfizer's haemophilia B gene therapy meets phase 3 primary endpoint
capsid (protein shell) and a high-activity human coagulation FIX gene. The purpose of this gene therapy is for those people living with haemophilia B, once treated, to be able to produce FIX via ...