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Published in 2020 at "BMJ Innovations"
DOI: 10.1136/bmjinnov-2020-000425
Abstract: Background Gene therapies have the potential to be a curative approach to a large number of genetic diseases. However, granting of a positive marketing authorisation does not equal patient access to therapy. Objectives The purpose…
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Keywords:
patient access;
therapy;
access gene;
gene therapies ... See more keywords