There are a couple of observations I had about the release that I posted on twitter. First to put in context of SOC I'm in the camp that believes its a tremendous advance and even with durability questions they will get meaningful commercial sales. 1-The PR was quite cheezy in what they did with the graphics! https://twitter.com/MauriceOnTW/status/1273269482065203203?s=20 Fortunately you can get higher res images from here https://twitter.com/MauriceOnTW/status/1273330596593504257?s=20 2-The four year high dose data mean 24.2/median 16.4 is a bit misleading (perhaps the reason for the power slide resolution?) as it includes just 6/7. If one goes by the 3 year 9 month reading looks to be about mean 20/median 12 https://twitter.com/MauriceOnTW/status/1273291002074345472?s=20 3-The data is not going to be reflective of commercial dose as Phase 3 interim data (what BMRN used to file on) produced very notably lower expression levels.
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