We don’t really know much for sure about the safety and tolerability of the 50mg dose.
Remember that the only study where we are using it is the current phase IIb study, and that is blinded so we don’t know which patients are getting that and which are getting 25mg.
It does seem that we’re not seeing any adverse reactions overall, so one would assume that the 50mg dose is pretty good in absolute terms, but we won’t really know the detail until we see unblinded safety data.
Once we have all the data in hand – safety and efficacy – we can make a decision about which dose to take forward. That will probably be something of a discussion with regulatory agencies too.
If both look good, it will be a nice problem to have.
I’m confident that the 50mg will be the superior dose.
We wait
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Ann: Phase IIb study of ATL1102 in DMD completes recruitment, page-195
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