Lindsey Shapiro, PhD, science writer —

Diminished production in the brain of a protein known as CHCHD2 may contribute to early neurodevelopmental changes that go on to drive Huntington’s disease, a study reports, with scientists poising the gene responsible for its production as a possible therapeutic target. CHCHD2 is involved in maintaining the workings of…

Clumps of the mutant huntingtin protein, which are the known cause of Huntington’s disease, poke holes in the membrane that surrounds and protects cells’ DNA-filled nucleus, according to preclinical research. These protein aggregates had tiny, protruding fibers that damaged the membrane, which is called the nuclear envelope. Lab-grown cells…

The investigational gene therapy AMT-130 is slowing disease progression for people with early-stage Huntington’s disease, as well as lowering levels of neurofilament light chain (NfL), a marker of nerve cell damage. That’s according to two-year data from two Phase 1/2 clinical trials: one in the U.S. (NCT04120493)…

More than three months of treatment with WVE-003 safely leads to significant reductions in mutant huntingtin — the protein that toxically accumulates in Huntington’s disease — for people with early Huntington’s, while preserving levels of the healthy version of huntingtin and trends of slower brain tissue loss and clinical…

The effects of Ingrezza (valbenazine) on Huntington’s disease-associated chorea grew increasingly stronger over the course of the three-month Phase 3 KINECT-HD clinical trial, according to a post-hoc analysis. A post-hoc analysis is one planned and conducted after the trial has been completed. “This post-hoc analysis of the…

Daily treatment with Ingrezza (valbenazine) led to sustained reductions in chorea — characterized by involuntary, unpredictable body movements — for nine months among the small group of Huntington’s disease patients simultaneously using antipsychotic medications in the Phase 3 KINECT-HD2 trial. In addition, improvements in chorea were also…

The brain of people with pediatric-onset Huntington’s disease show impaired transport of blood sugar, or glucose, and low levels of a cellular machinery involved in glucose metabolism, a small study showed. Many of these abnormalities were observed in comparisons not only with adults without Huntington’s, but also with people with…

Note: This story was updated Jan. 24, 2024, to correct that Takeda is not supporting WVE-003’s development. Wave Sciences is the treatment’s sole developer; Takeda has a codevelopment and co-commercialization option. New data from a Phase 1/2 trial of WVE-003, a therapy targeting the mutant huntingtin protein in people…

In 2023, Huntington’s Disease News brought you the latest in scientific breakthroughs and treatment developments related to Huntington’s disease. To recap what interested our readers most this year, here are the top 10 most-read articles, with a brief description of each. We look forward to serving as a…

The contents of Ingrezza (valbenazine) can be taken from their capsules and crushed before being sprinkled on soft foods or given via a feeding tube to Huntington’s disease patients who have difficulty swallowing whole capsules, a preclinical study showed. A Neurocrine Biosciences’ oral therapy, Ingrezza…