News

Up to four years after a single dose of the experimental gene therapy ifezuntirgene inilparvovec (formerly AMT-130), people with Huntington’s disease who received the high dose showed slower disease progression than would be expected without treatment. That’s according to data from two long-term Phase 1/2 clinical trials, one…

Time-restricted eating (TRE), a form of intermittent fasting that limits daily calorie intake to a six-to-eight-hour window, may help slow the progression of Huntington’s disease, according to results from a three-month pilot study. Study participants were able to maintain their body weight while showing reductions in disease severity measures…

In final 15-month study data, people with early-stage Huntington’s disease treated with SKY-0515, Skyhawk Therapeutics’ investigational oral therapy, showed improvements in function, movement, and cognition. That’s according to final results from Skyhawk’s Phase 1/2 clinical program, which included the registered Phase 1 trial (ACTRN12624000602527). The study included…

An early and progressive loss of a protein that helps maintain myelin, or the protective sheath around nerve fibers, may contribute to white matter damage in Huntington’s disease, a study suggests. In a mouse model of Huntington’s, levels of the myelin-associated glycoprotein (MAG) began falling before the loss of…

uniQure has submitted an application to the U.S. Food and Drug Administration (FDA) seeking accelerated approval of AMT-130, the Netherlands-based company’s gene therapy candidate for Huntington’s disease. A similar request for marketing authorization in the U.K. has been made to the nation’s Medicines and Healthcare products Regulatory…

A real-world study shows that the approved chorea treatment Austedo (deutetrabenazine) reduces involuntary movements in adults with Huntington’s disease and is generally well tolerated in clinical settings. According to the researchers, most patients experienced a decrease in the severity…

A speech index score built from smartphone voice recordings tracked the severity of Huntington’s disease and matched closely with standard clinical assessments and brain scan measurements, according to a new study from China. The index score, derived from measures of loudness variation, mispronunciation rate, pitch strength, and speech timing,…

Soma and Neurite Density Imaging (SANDI), an imaging technique that uses MRI scans to capture microstructural features of the brain, may be a useful tool for tracking the progression of Huntington’s disease, a study suggests. Huntington’s patients’ SANDI data, together with age, could explain up to 63% of shrinkage…

Motion data collected by a smartwatch may offer a more objective way to monitor worsening involuntary movements in people with Huntington’s disease than clinic visits alone, a study found. Researchers in the U.K. and Switzerland developed a digital tool to estimate the severity and progression of chorea, a hallmark…

A novel gene-editing approach that’s designed to alter the huntingtin protein so it’s less prone to forming the toxic clumps that drive Huntington’s disease led to better motor function and less brain damage in a mouse model of the disease, a study shows. “Our results suggest a new way…