@waynesworld
Wayne.. did you sense a very different tone at PPMD this year?
In previous years, 99% of the focus on drug development was on Dystrophin restoration strategies. They were really hoping Gene Therapy was a CURE!
This year there was a huge shift towards the downstream effects and in particular Inflammation, with emphasis on Combination therapies.
They seem to have held up the white flag on Dystrophin being the big saviour. You could sense the disappointment and almost a resetting of expectations.
ANP and ATL1102 got multiple mentions across a range of presentations. The profile shift was palpable.
The moons seem to be aligning for us and we can expect this to increase over the coming year. Next year at PPMD, we will have trial results in hand.
Will the realisation that Dystrophin is not the be all and end all, I wonder what the discussion in the Sarepta boardroom is?
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