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Sanofi, with positive data in hand, to ask for approval of longer-lasting hemophilia therapy

Sanofi and its development partner, the Swedish drug company Sobi, said Wednesday they plan to submit an experimental, longer-lasting hemophilia treatment for approval now that …

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Fresh data show UniQure’s hemophilia gene therapy appears to hold up

Gene therapies have the potential to essentially fix many diseases. Yet, those currently in development are administered just once, raising questions about whether their effects …

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gene therapy

Pfizer gene therapy research delayed by trial changes, safety questions

In both hemophilia and Duchenne, unexpected findings have led Pfizer to redraw its development plans. The company recently paused patient screening and dosing in a …

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