Articles with the topic: splice modulators

Two birds, one stone: HTT-lowering drugs also target CAG expansions

Two birds, one stone: HTT-lowering drugs also target CAG expansions

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.

Dr Sarah HernandezMay 21, 2024

Updates from PTC Therapeutics and uniQure on their huntingtin-lowering trials

Updates from PTC Therapeutics and uniQure on their huntingtin-lowering trials

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.

Dr Leora Fox and Dr Rachel HardingJune 21, 2023

Disappointing news from Novartis about branaplam and the VIBRANT-HD trial

Disappointing news from Novartis about branaplam and the VIBRANT-HD trial

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.

Dr Rachel HardingDecember 09, 2022