The outcomes of the SELECT-HD clinical trial are out, with encouraging news for this allele-selective huntingtin lowering therapy
PTC Therapeutics shared 12 month data from the PIVOT-HD trial, testing the oral HTT lowering drug PTC-518. While designed to assess safety, they shared encouraging results that the drug showed promising signs for biomarkers and some clinical metrics.
Data from GENERATION HD1, the Phase 3 clinical trial testing the huntingtin-lowering drug tominersen, have just been published in a scientific journal. The trial ended a while back, so why is this an important milestone, and what’s next?
The HD pipeline is rich and varied. Let's talk about some out-of-the-box approaches for developing drugs for HD that don't involve huntingtin lowering.
The FDA has approved valbenazine, also known as INGREZZA, as a treatment for the movement symptoms of Huntington’s disease
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.
The phase 3 trial missed its primary endpoint of slowing loss of function in Huntington’s disease
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.