News

The European Medicines Agency (EMA) has agreed to review Prilenia Therapeutics’ application seeking approval of pridopidine, an oral therapy candidate for adults with Huntington’s disease, the company announced. The regulatory application is based on safety and efficacy data from pridopidine’s clinical development program, which showed the therapy…

Examining the amounts of certain microbes in the gut and levels of specific metabolites in the blood may help distinguish people with and without Huntington’s disease with 100% accuracy, a study suggested. “This study determined crucial functional gut microbiota and potential biomarkers associated with [Huntington’s development], providing new insights…

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…

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…

A high level of education, low to moderate alcohol intake, not smoking, and controlling one’s weight may slow Huntington’s disease progression among those who’ve yet to have symptoms, a study shows. “Reducing modifiable risk factors for [Huntington’s] is one way to support the presymptomatic population,” which is made up…

Levels of glial fibrillary acidic protein (GFAP), a marker of damage to specific neuron-supporting cells, in the fluid surrounding the brain and spinal cord increase with the progression of Huntington’s disease, a study showed. Higher GFAP levels in the cerebrospinal fluid (CSF) were also significantly associated with more severe…

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,…

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…