Marisa Wexler, MS,  senior science writer—

LETI-101, an experimental gene-editing therapy for Huntington’s disease, showed promising proof-of-concept effects in mouse and cell models by reducing levels of the disease-causing mutant huntingtin (HTT) protein, while preserving HTT’s healthy version. In nonhuman primates, the therapy showed good safety and distribution across brain regions profoundly impacted by Huntington’s. The…

Specific genes involved in repairing mismatched DNA contribute to the development of Huntington’s disease by driving fast somatic CAG repeat expansion — a phenomenon in which Huntington’s-causing mutations grow, or expand, during a person’s lifetime, scientists report. Their study, done in a mouse model of Huntington’s, showed that targeting…

In specific brain cells, the mutation that causes Huntington’s disease grows more and more unstable as years go by, according to a study by U.S. researchers that offers new clues into the generally late onset of the neurodegenerative condition. The findings help to explain why people with Huntington’s don’t…

Abnormalities in a protein called TDP-43, which is known to play a key role in the development of amyotrophic lateral sclerosis (ALS), may also underly Huntington’s disease, a study found, meaning strategies being explored as treatments for ALS might also be useful for treating Huntington’s. “Drugs developed to…

Throughout 2024, the team at Huntington’s Disease News covered the latest developments in research and news related to Huntington’s disease. Here we’ve compiled a list of the top 10 most read stories, each with a brief summary. We look forward to continuing to serve the Huntington’s community throughout…

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…

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…

Being physically active is significantly associated with a delayed age of the onset of Huntington’s disease and spending more time watching television is significantly linked to an earlier age of onset. These are the findings of a study that analyzed genetic variants linked with different levels of physical activity…

Ingrezza Sprinkle, a new formulation of Ingrezza (valbenazine) that’s designed to be easier to swallow, is now available in the U.S, Neurocrine Biosciences, which markets the therapy, announced. Ingrezza Sprinkle comes in capsules that are meant to be opened and the contents sprinkled over soft foods like yogurt,…