Gene therapy in hemophilia advances with big drops in patient bleeding rates – STAT
STAT |
Gene therapy in hemophilia advances with big drops in patient bleeding rates
STAT t's still early days for efforts to develop gene therapies against hemophilia, but a handful of patients treated with these one-time, potentially curative treatments have now been followed long enough to start measuring clinically relevant outcomes … BioMarin lays out its PhIII strategy for its closely-watched gene therapy for hemophilia A BioMarin's gene therapy candidate for hemophilia A shows durable treatment effect in mid-stage study |
