Articles with the topic: huntingtin-lowering

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

Steady progress from uniQure - promising data to end the year

Steady progress from uniQure - promising data to end the year

uniQure ushered in the end of the year by releasing some promising data from their huntingtin-lowering gene therapy trials

Dr Leora FoxDecember 20, 2023

Could halting CAG expansions be a new treatment for HD?

Could halting CAG expansions be a new treatment for HD?

The gene MSH3 is getting a lot of attention in HD research lately. New findings suggest MSH3 lowering could halt CAG repeat expansions, offering a new therapeutic avenue.

Dr Sarah HernandezOctober 05, 2023

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

Hunting for balance: how the huntingtin protein compensates in HD

Hunting for balance: how the huntingtin protein compensates in HD

Researchers look at the cause and effect of various forms of the HTT protein. They find both expanded and unexpanded HTT contribute to brain cell communication and the brain has an amazing capacity to compensate for changes related to disease

Dr Sarah Hernandez and Dr Leora FoxApril 05, 2023

Roche Phase II GENERATION HD2 study underway

Roche Phase II GENERATION HD2 study underway

Roche released a community letter in early 2023, to share that their Phase II clinical trial to study the huntingtin-lowering drug, tominersen, is now underway. In this article, we summarise the latest news about this huntingtin-lowering drug.

Dr Rachel HardingFebruary 14, 2023

uniQure gets the green light to resume testing HD gene therapy

uniQure gets the green light to resume testing HD gene therapy

Following a 3-month pause in enrollment due to concerns about side effects, uniQure shared the good news that their trial of the HD gene therapy AMT-130 will continue as planned, with new safety measures in place.

Dr Leora FoxNovember 03, 2022

Update on the PTC Therapeutics PIVOT-HD Trial

Update on the PTC Therapeutics PIVOT-HD Trial

Last week, PTC Therapeutics released a statement sharing that recruitment of participants into the US arm of the PIVOT-HD trial has been paused. In this article, we will lay out exactly what is known and what this announcement means.

Dr Rachel Harding and Dr Leora FoxNovember 03, 2022

Forward momentum for Roche and Wave in latest news about huntingtin-lowering trials

Forward momentum for Roche and Wave in latest news about huntingtin-lowering trials

Two companies developing medicines for Huntington’s disease announced news about their huntingtin-lowering drugs. Let’s talk about the history and the caveats surrounding these hopeful pieces of news.

Dr Jeff Carroll and Dr Leora FoxSeptember 30, 2022