In a surprising twist, oral HTT-lowering drugs also slow somatic expansion in the HTT gene. A new study that used cells in a dish for this fortuitous discovery identified the gene PMS1 as a key player in the slowing of CAG expansions.
Both PTC Therapeutics and uniQure have shared updates from their respective clinical trials, testing different huntingtin-lowering approaches. We explore the data presented from both of these studies and what this means for HD family members.
Novartis have announced that they are ending development of the drug branaplam in Huntington’s disease. Here, we review this latest news and its impact on the HD community.