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

The pharmaceutical company Novartis has released a community update which announces that they are ending development of branaplam, a huntingtin lowering drug, for possible treatment in Huntington’s disease (HD). This news comes following recent bad news about side effects of branaplam in HD patients, being tested in the VIBRANT-HD clinical trial, dosing of which was […]

uniQure gets the green light to resume testing HD gene therapy

In August 2022, uniQure announced a pause in new recruitment for their trial of AMT-130, an HD gene therapy delivered via brain surgery. The decision was made after 3 out of 14 participants who had received a high dose of the drug experienced serious side effects after the procedure. The trial’s Data Safety Monitoring Board […]

Update on the PTC Therapeutics PIVOT-HD Trial

Recruitment of participants into the US arm of the PTC Therapeutics PIVOT-HD trial has been paused. Since this announcement, there have been a lot of different (and confusing!) headlines about the pause in recruitment. In this article, we will lay out what is going on and what this announcement means. What is the aim of […]

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

In the past week or so, during and following a big HD research conference, two companies developing medicines for Huntington’s disease announced news about their huntingtin-lowering drugs. First, the pharmaceutical company Roche announced plans for a new clinical trial of tominersen. Then, the genetic medicines company Wave Life Sciences shared early data showing that its […]

Focusing in on fibrils; scientists give us a glimpse of huntingtin protein clumps

A group of scientists from the EPFL in Lausanne, Switzerland have published a paper in the Journal of the American Chemical Society, describing clumps made up of a fragment of the huntingtin protein. A word that’s commonly used to describe these is “aggregates.” Using very powerful microscopes, the team was able to zoom in and […]

Hereditary Disease Foundation (HDF) conference 2022 – Day 4

DNA repair and CAG repeat instability The effect of HTT lowering on CAG repeat expansions Welcome to last day of the @hdfcures conference! We’ll only be sharing a few talks from today’s sessions, which focus on DNA repair. The first is from HDBuzz’s very own Jeff Carroll! Jeff will be sharing his work on HTT […]