The following is excerpted from the question-and-answer section of the transcript.
(Questions from industry analysts are provided in full, but answers are omitted - download the transcript to see the full question-and-answer session)
Question: Jon Wolleben - JMP Securities LLC - Analyst
: Hey, good afternoon and congrats on the data, pretty compelling stuff. Couple of questions, wondering if you have any sense from data in the
older children or whether mechanistically, when responses would plateau. Three years, that was quite a long time, but I can't imagine that things
will get better forever, but do you guys have any inkling what that could look like?
Question: Jon Wolleben - JMP Securities LLC - Analyst
: Got it. And safety looks to be improving. I know you guys gave treatment-related anaphylaxis. Wondering if you could comment at all on unrelated
anaphylaxis and unrelated epinephrine use, like in the context of an accidental exposure.
Question: Jon Wolleben - JMP Securities LLC - Analyst
: Okay. And then maybe last one for me, and I'll jump back in the queue. I noticed in the release that the label in, label out, which I think will be
tremendously helpful in the real world. You started that 90-day counter after the first month. And I was wondering if you could walk through a
little bit about the rationale for that and what this actually will look like.
Question: Jon Wolleben - JMP Securities LLC - Analyst
: Makes perfect sense. Alright, thanks guys.
Question: Jon Wolleben - JMP Securities LLC - Analyst
: I'll take advantage of the time. How did you determine 20 hours for the cutoff? And did you look at that as a continuous parameter as well?
Question: Jon Wolleben - JMP Securities LLC - Analyst
: Yes. And then, last one for me, if you could just provide an update on kind of activities necessary to get the comfort trials up and running on
schedule.
Question: Jon Wolleben - JMP Securities LLC - Analyst
: Alright, thanks again.
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