Haemophilia
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EMA guidance · 2024-05-31New gene therapy treatment for haemophilia B
The EMA announced that the gene therapy Durveqtix for haemophilia B has received PRIME designation to support its development and potential …
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EMA guidance · 2018-11-30Data from patient registries to replace clinical trials in previously untreated haemophilia patients
EMA discusses using data from patient registries as a potential alternative to traditional clinical trials for previously untreated haemophi…