News

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…

A team led by scientists at a U.K. research organization has created a questionnaire to measure quality of life among people with manifest Huntington’s disease, meaning those who are experiencing the hallmark motor symptoms that characterize the genetic disorder. Dubbed the Huntington’s Disease Manifest Quality of Life measure (HD-mQoL),…

A global clinical trial testing SKY-0515, Skyhawk Therapeutics’ daily oral therapy for Huntington’s disease, will soon start recruiting adults with the genetic condition in the U.S., Canada, and the U.K. Regulatory authorities in all three countries have granted permission to open sites for the worldwide portion (004-WW,…

An early-stage clinical trial designed to test a novel stem cell therapy — one designed to replace lost or damaged nerve cells in the brain — in people with Huntington’s disease is now recruiting adults with the genetic condition at its single site in California. The Phase 1/2 trial, dubbed…

An oral compound now being tested as a potential treatment for several neurodegenerative conditions was shown, in multiple laboratory models, to reduce the toxic protein clumps in nerve cells that drive Huntington’s disease. These findings suggest that the compound, called Anle138b, may also be a possible therapeutic…