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Published in 2020 at "Haemophilia"
DOI: 10.1111/hae.14218
Abstract: Early‐stage gene therapy (GT) clinical trials are demonstrating exciting results for persons with haemophilia (PWH), with the first products possibly licenced over the next few years for haemophilia A and B. These new treatments offer…
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Keywords:
uncertainty;
gene therapy;
value gene;
reimbursing value ... See more keywords