Comment on Breakthrough in Huntington’s disease treatment shows unprecedented results for patients
SaveTheTuaHawk@lemmy.ca 1 day ago
This was not a clinical trial, it was a safety study of only 11 treated. No controls.
CEO of UniQure already dumped all his stock options.
verdi@feddit.org 1 day ago
It was a phase I\II trial. Dafuq u on about?
Phase I\II determine efficacy and safety profiles.
In this case they are proceeding with a new cohort where the administration will be concurrent with immune suppression to counteract inflammation in the pos-op phase.
They will likely apply for a phase III after since this is an orphan drug.