I joined the Biomarin conference this evening. Not much more info than the press release but here are a few snippets that I learned. 1/7 
Biomarin phase 3 steroid use. Number of patients who needed steroids not mentioned. No mention of steroid adverse events. They said they just completed recruitment of a study that uses prophylactic steroids. 2/7
I believe that in #hemophilia gene therapy studies the adverse events due to steroids should be mentioned. These are high dose steroids for several weeks and adverse events are common. 3/7
Lots of questions on durability of the Biomarin phase 3 data on gene therapy in #hemophilia. Expected efficacy of 6-7 years mentioned. It is important that patients realise that (at least in #haemophilia A), at present, gene therapy is not a therapy for life. 4/7
Biomarin place their confidence of durability of their gene therapy in #hemophilia on extrapolation from the data of their phase 1/2 6e13 cohort who are now in year 5 but that cohort has only 7 patients. 5/7
No data on variability of response were shown. As a patient going for this therapy you would want to know "what are my chances I will achieve a FVIII level that will allow me to stop prophylaxis?" 6/7
Biomarin are anticipating approval of their gene therapy in #hemophilia in Europe in the second half of 2022. 7/7
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