News

Data on medicines for Huntington’s psychiatric symptoms lacking

Mental health problems are common in Huntington’s disease, but there isn’t much data on patients’ use of psychiatric medications, a review study reports. “Although [neuropsychiatric problems] in [Huntington’s disease] are common, hardly any clinical trials have addressed their treatment,” the study’s researchers wrote. “As a result, evidence that…

Wave planning potentially pivotal trial of WVE-003 in patients

Wave Life Sciences is making plans to launch a potentially pivotal Phase 2/3 clinical trial in the U.S. to test its therapy candidate WVE-003 in people with Huntington’s disease. Pivotal trials are those whose data, if positive, are meant to be used to support applications seeking a treatment’s…

LETI-101 lowers mutant HTT protein in preclinical models

LETI-101, an experimental gene-editing therapy for Huntington’s disease, showed promising proof-of-concept effects in mouse and cell models by reducing levels of the disease-causing mutant huntingtin (HTT) protein, while preserving HTT’s healthy version. In nonhuman primates, the therapy showed good safety and distribution across brain regions profoundly impacted by Huntington’s. The…

DNA repair genes contribute to Huntington’s, mouse study suggests

Specific genes involved in repairing mismatched DNA contribute to the development of Huntington’s disease by driving fast somatic CAG repeat expansion — a phenomenon in which Huntington’s-causing mutations grow, or expand, during a person’s lifetime, scientists report. Their study, done in a mouse model of Huntington’s, showed that targeting…

Early neurodegeneration biomarkers linked to CAG repeat growth

Subtle biomarkers of neurodegeneration are evident decades before the onset of overt Huntington’s disease symptoms in people with disease-causing mutations, a study found. Somatic CAG repeat expansions — a phenomenon in which Huntington’s-causing mutations grow, or expand, during a person’s lifetime — were associated with these neurodegenerative biomarkers.