By replacing defective or missing genes, scientists aim to cure life-long and highly debilitating diseases such as haemophilia.
Around 69,000 people in Europe have Haemophilia A. Patients with moderate to severe Haemophilia require intravenous replacement therapy every 3-7 days to prevent bleeding incidents.
What is the potential breakthrough?
What is the potential impact on Europe’s healthcare systems?
Download the full IQVIA/EFPIA Pipeline Review 2021
#WeWontRest until we make treatments for rare disease less rare
Disease never takes a break, so neither do we. We won’t rest until people around the world see a brighter tomorrow.Read more