News

The U.S. Food and Drug Administration (FDA) has agreed that results from two ongoing Phase 1/2 clinical trials, compared with an external control group of untreated patients, might be sufficient to support an application seeking accelerated approval of AMT-130 for Huntington’s disease, the treatment’s developer said. Key measures…

Beta-blockers, a class of medicines often used to manage heart and blood pressure issues, may slow the onset and progression of Huntington’s disease, according to data from Enroll-HD, the world’s largest observational study in Huntington’s. “Given that there are no disease-modifying agents for HD [Huntington’s disease], the possibility that…

Sarepta Therapeutics has entered a global licensing and collaboration agreement with Arrowhead Pharmaceuticals covering Arrowhead’s therapy candidates, including ARO-HTT, an experimental treatment for Huntington’s disease. Sarepta will obtain exclusive global rights to ARO-HTT and other preclinical programs, as well as clinical and discovery-stage programs for rare genetic…

Sage Therapeutics is discontinuing the development of dalzanemdor after the investigational therapy failed to improve cognitive abilities relative to a placebo among people with Huntington’s disease enrolled in a Phase 2 clinical trial. Statistically significant gains in cognition with treatment was the main goal of the…

Ingrezza (valbenazine), an approved treatment for chorea, or uncontrolled movements, related to Huntington’s disease, shows similar effectiveness among patients irrespective of factors like sex, age, or disease severity, according to a new analysis of data from a Phase 3 clinical trial. Moreover, a separate data analysis from…

Researchers have developed a protein-like polymer — a large, string-like molecule composed of small repeating building blocks — that was able to slow the onset of Huntington’s disease in a mouse model. In the mice, the protein-like polymer prevented the clumping of misfolded huntingtin protein that drives nerve cell…

Alnylam Pharmaceuticals has launched a Phase 1 clinical trial to test the safety and pharmacological properties of its treatment candidate ALN-HTT02 in adults with Huntington’s disease, the company announced as part of a financial update. “We [have] made great strides with our pipeline, … initiating a Phase 1…

Higher levels of an enzyme called GST02 in the most affected neurons in Huntington’s disease could underlie early increases in the brain-signaling chemical dopamine that are believed to drive Huntington’s motor symptoms, according to a mouse study. Scientists had previously uncovered that a deficiency in a signaling pathway called…

Blood levels of neurofilament light protein (NfL) in people who carry Huntington’s disease-causing mutations, but aren’t showing symptoms, can identify those who will develop the disease in the next decade. These are the findings of a 14-year study that also showed that greater increases in blood NfL levels, a…

The U.S. Food and Drug Administration (FDA) is supporting financially a study into how well wearable sensors measure two early signs of Huntington’s disease, which could be of use to clinical trials of potential treatments. The funding comes from the agency’s Digital Health Technologies for Drug Development…