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.
COVID-19 update: what does it mean for HD families, how does it impact HD research, and how has it changed the way science works?
Wave Life Sciences announces that its antisense drug WVE-120102 has lowered mutant huntingtin protein in cerebrospinal fluid, but investors seem disappointed. Rather confusing – what do we know for sure?
UniQure announces key details of its planned trial to assess the safety and ability of AMT-130 gene therapy to lower the problematic huntingtin protein using a ‘single-shot’ virus delivery system.
This fall sees exciting announcements from a number of companies focused on novel Huntingtin Lowering technologies, including Wave, PTC and Voyager
The LEGATO-HD trial of laquinimod did not slow progression of Huntington's disease. Here's the lowdown.
Amazing news from Ionis and Roche! HTTRx drug successfully lowers harmful huntingtin protein in spinal fluid