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Biomarin has just announced their year 2 phase 3 (134 patients) ValRox gene therapy in #hemophilia results. Yes on a Sunday evening! Stable and durable bleed control but the FVIII levels are falling significantly. tinyurl.com/2p92ksj8 1/4 
Key questions: How many patients needed steroids, what was the variability in levels (and how many had levels <10% and <5%), how long is the response predicted to last? #hemophilia #genetherapy #ValRox 2/4
Considering the mean FVIII levels, it is not surprising that stable and durable annual bleed control was observed. I would expect not many patients in the <10% group. Loss of ABR control would only be expected when the levels fall below 10%. #hemophilia #genetherapy 3/4
