😂

Any thoughts on ptct data just released now?

I think knockdown mHTT pre-symptom would be where the field should head in the future. But how do you design such trial? At what age and how much knockdown?
But as of now, targetting exon-1a should be in the focus.

Hey Dirk, isnt Handsaker’s findings kinda validate the point of targetting exon1 ? Extensive somatic expansion -> Onset where exon-1a leads to cellular toxicity.