WOW! That's great news! They said previously that they only considered one of those 5 (which would have been strange) but now all 5 are approved. I hope they find a strategy to move forward with the rest of residual function splice mutations, there are so many different ones. It seems that they didn't use in vitro testing to approve these and I have not seen any published results of in vitro testing of potentiator efficacy on splicing mutations so I wonder if in vitro results are actually unreliable when it comes to splicing defects?
