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Prof Haluk Topaloğlu

Position Statement: Emerging genetic therapies for rare disorders at high cost, cannot realistically address the global burden of disease


Emerging genetic therapies for rare disorders at high cost, cannot realistically address the global burden of disease - Stakeholders must develop new pathways to ensure safe, fair and sustainable provision of such therapies A disease or disorder is defined as rare in Europe when it affects fewer than 1 in 2000 people and based on the Food and drug administration (FDA) definition from the US, when it affects less than 200,000 at any one time. Genetic factors contribute to the etiology in 80% of those with rare diseases, and other contributing factors, including infection, account for the remaining the cases....
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