News

Treatment with antioxidant compounds called fullerenols showed neuroprotective effects in a fruit fly model of Huntington’s disease, particularly at early ages, according to a new study. These findings add to a number of studies highlighting the role of oxidative stress in Huntington’s, and suggest that fullerenols may have…

Spark Therapeutics and Neurochase have joined forces to develop Neurochase’s proprietary delivery technology for gene therapies that target neurodegenerative diseases, such as Huntington’s disease. The technology, called Convection Enhanced Delivery (CED), allows for a controlled delivery of gene and other therapies directly into specific brain regions with the…

In Huntington’s disease, neurodegeneration in a brain region called the striatum differs not only across different types of neurons, but also across distinct striatal compartments, according to a study of brain samples from a Huntington’s patient and mouse models. The striosomes, the striatal compartment involved in regulating mood, was more…

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…