Breakthrough in gene therapy on Haemophilia B
An early UK / US study indicates a “potentially life-changing" gene therapy for people with Haemophilia B.
According to the study, a single injection could be enough for Haemophilia B sufferers to no longer need medication.
UK medical researchers successfully treated six patients suffering from the blood-clotting disease by injecting them with the correct form of a defective gene. Four of them could no longer need their medication.
This success is the result of several minor improvements developed over many years by different groups of researchers.
- Read the NY Times article
- Read about EURORDIS and EU advanced therapy regulation
- Read about EURORDIS and the COMP (Committee for Advanced Therapies at the European Medicines Agency)
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