News

Reduced levels of oxytocin — a natural hormone produced in the brain’s hypothalamus that also works as a chemical messenger — in the cerebrospinal fluid (CSF) were found in Huntington’s disease patients with cognitive and social impairments, a new study shows. The findings support oxytocin levels as a biomarker of impaired…

Neuron-targeted treatment based on zinc-finger DNA-binding proteins — called ZFPs — was found to lower brain levels of mutant huntingtin (mHTT) protein in a Huntington’s disease mouse model. The zinc-finger proteins rescued behavioral deficits and extended survival in these mice, the study demonstrated. ZFPs are among the most abundant…

The U.S. Food and Drug Administration (FDA) has agreed to review Neurocrine Biosciences’ supplemental new drug application (sNDA) for valbenazine to treat chorea caused by Huntington’s disease. “This sNDA filing advances our effort to bring a potential new treatment option to the many thousands of people experiencing chorea associated with…

Researchers have developed a new therapy that reduced the toxic buildup of the mutant huntingtin (HTT) protein — the hallmark of Huntington’s disease — in lab-grown neurons from Huntington’s patients and in a mouse model of the disease. The CRISPR-based therapy was designed to specifically target the HTT gene’s messenger…

Branaplam (LMI070), an experimental oral therapy for Huntington’s disease that’s currently in early clinical trials, reduces levels of the mutant huntingtin protein by interfering with a process called mRNA splicing, a new study reveals. “Here, we delineated the mechanism of action of Branaplam,” researchers wrote. The study, “…

Abnormal red blood cells, known as acanthocytosis, were identified in four unrelated Chinese adults with Huntington’s disease — undermining researchers’ attempts to differentiate the neurodegenerative disorder from another similar genetic disease. Researchers had expected that the presence or absence of such abnormally shaped red blood cells would help…

Long-term treatment with Austedo (deutetrabenazine) safely led to sustained reductions in chorea for people with Huntington’s disease, including those who had switched from treatment with Xenazine (tetrabenazine). That’s according to about three years of follow-up data in the Phase 3 ARC-HD trial (NCT01897896), a study jointly conducted…

A data safety monitoring committee has recommended that enrollment of the highest dosing group resume in the European Phase 1/2 trial of AMT-130 in people with Huntington’s disease, but with additional safety measures in place. UniQure, the investigational treatment’s developer, announced in August that enrollment in…

A greater number of CAG repeats within the HTT gene — the genetic cause of Huntington’s disease — is linked to earlier disease onset and death, but is not an independent predictor of mortality in people with the neurodegenerative disease, a study shows. These findings suggest that “non-genetic factors…

The Hereditary Disease Foundation (HDF) is recognizing two scientists, Steven Finkbeiner and Natalia Barbosa, for their leadership in Huntington’s disease research. Both will speak at the organization’s 2022 Virtual Gala, set for Nov. 10 starting at 7 p.m. ET. The free, hourlong event will cover…