Forest Ray PhD,  —

Brain-derived neurotrophic factor (BDNF) may not be the biomarker for Huntington’s disease that it was previously thought to be, a recent study reported. The progressive loss of BDNF, a factor that helps neurons grow and survive, associates with Huntington’s progression in mouse disease models, and past studies found it at reduced…

The first two patients have been enrolled in the high-dose group of a U.S. clinical trial testing the safety and efficacy of AMT-130 as a potential gene therapy for Huntington’s disease. “We are very pleased with the progress that we continue to make in this Phase 1/2 clinical trial and…

Metabolites seen as potential biomarkers of Huntington’s disease — called kynurenine pathway metabolites — showed little evidence of Huntington’s-specific activity in a recent study. While researchers could not entirely rule out kynurenine pathway metabolites as possible treatment targets, their results suggest that the disease is not well represented by the…

Stalled protein-building may underlie Huntington’s disease, according to data from a recent study. The findings showed that the mutated huntingtin protein, which is at the heart of the disorder, slows down ribosomes, the small cell machinery structures responsible for building new proteins. According to investigators, knowing this and understanding…

Wearable devices show considerable promise in helping diagnose people with Huntington’s disease, but the studies evaluating them need to be standardized to maximize their potential benefit to patients, a review found. That review determined that these devices can distinguish people with and without Huntington’s, and between symptomatic and pre-symptomatic…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

The Hereditary Disease Foundation‘s (HDF) has announced the first winner of its Nancy S. Wexler Young Investigator Prize, supporting excellence and creative thinking in Huntington’s disease research. The inaugural award went to Osama Al-Dalahmah, a neuropathology instructor in the department of pathology and cell biology…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

Microbes in the gut of people with Huntington’s or likely to develop this disease appear to differ from healthy peers, opening the door to potential new biomarkers and therapeutic targets, according to a recent study. The study, “Gut dysbiosis in Huntington’s disease: associations between gut microbiota, cognitive…