Margarida Maia, PhD,  science writer—

Pediatric-onset Huntington’s disease is more severe than its adult-onset form, with faster and more extensive shrinkage of brain regions involved in movement, an imaging study has found. Atrophy, or a loss in brain volume, “was remarkable and more severe” in patients with pediatric-onset disease compared with adult-onset Huntington’s, the…

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…

Loqus23 Therapeutics has raised 35 million pounds (about $45 million) in financing to advance its lead oral small molecule candidate for Huntington’s disease through preclinical development and early clinical studies, which are anticipated in 2026. The series A financing round was led by Forbion, a life sciences…

Unlike its healthy counterpart, the mutated version of the huntingtin protein that drives Huntington’s disease may keep nerve cells from repairing their damaged DNA, causing them to die, a study showed. Specifically, the mutated protein failed to stimulate the production of poly ADP-ribose, known as PAR — a molecule…

Vico Therapeutics’ investigational therapy VO659 was found to lower levels of the disease-causing mutant huntingtin (HTT) protein — known as mHTT — in the cerebrospinal fluid (CSF), the liquid that surrounds the brain and spinal cord, of people with Huntington’s disease. Additionally, treatment was not associated with an…

Researchers in the U.S. have created the first brain-like organoids, or mini-brains, that incorporate a working blood-brain barrier (BBB), which is the membrane that regulates which circulating substances can reach the brain and spinal cord, and that typically challenges the entry of medications. Called a BBB assembloid, the human model…

uniQure expects to begin planned discussions with the U.S. Food and Drug Administration (FDA) in the coming months to outline the future steps for testing its gene therapy candidate AMT-130 for early Huntington’s disease, and seeking regulatory approval of the treatment. In its interactions with the FDA, expected to…

Ayana Bio was awarded $300,000 from the National Institutes of Health (NIH) to sample saffron — the world’s most expensive spice — grown in a lab and identify which of its compounds offer the strongest neuroprotective effects for Huntington’s disease. The Boston-based company will use the grant,…

The U.S. Food and Drug Administration (FDA) has approved Neurocrine Biosciences’ oral granules formulation of Ingrezza (valbenazine) — called Ingrezza Sprinkle — for treating chorea, or involuntary movements, in adults with Huntington’s disease. The new formulation, taken once per day, is designed to provide an easier administration option…

A type of brain nerve cells called layer 5a pyramidal neurons — especially those projecting into the striatum, a brain region greatly affected in Huntington’s disease — are lost early in the course of Huntington’s, a study found. Excessive CAG repeats in the HTT gene, the cause of…