The first group of 10 participants have been dosed in uniQure’s clinical trial of an HD gene therapy, and three new manuscripts describe safe, widespread huntingtin lowering in animals.
Wave Life Sciences shared the disappointing news that their two ASOs in Phase 1/2 trials in HD patients did not successfully lower mutant huntingtin.
Disappointing news from Roche and Ionis; the phase III Tominersen huntingtin-lowering trial has been halted early
Read our summary of the latest updates from the EHDN Plenary Meeting 2020
The 2020 virtual HSG conference HD in Focus continues on Day 2 with a focus on clinical trials and drugs in development.
Read our breakdown of some of the talks and presentations at day 1 of the Huntington Study Group (HSG) 2020 annual conference: HD in focus
While developing a drug called branaplam for patients with SMA, the pharmaceutical company Novartis discovered that it could hold promise for people with HD. The FDA has granted a special status called Orphan Drug Designation.
The SIGNAL study did not meet its key clinical goals for #HuntingtonsDisease to slow or improve HD symptoms, but the results are still informative for the HD community and other fields.
We know that HD-related changes can occur many years before symptom onset, but how early do those changes begin? A team of researchers set out to determine that with a new comprehensive study in pre-manifest HD young adults.