Lindsey Shapiro, PhD,  science writer—

A data safety monitoring committee has recommended that enrollment of the highest dosing group resume in the European Phase 1/2 trial of AMT-130 in people with Huntington’s disease, but with additional safety measures in place. UniQure, the investigational treatment’s developer, announced in August that enrollment in…

A single injection of Wave Life Science‘s experimental therapy WVE-003 was found, at either of the two lowest tested doses, to lead to reductions in the mutant huntingtin (mHTT) protein in people with Huntington’s disease — while leaving the healthy version of the protein unaffected. That’s according to…

Social support for children of a parent with Huntington’s disease from someone other than a caregiver is important in helping them manage stress and adversity, but there are obstacles to obtaining that support, a study has found. People who’ve lived with a parent with Huntington’s reported that, while social…

A Phase 2 trial of pepinemab failed to significantly improve certain cognitive abilities in people with prodromal to early Huntington’s disease, not meeting a primary study goal. But data indicated that diagnosed patients in early disease stages, those with existing cognitive and functional impairments at the study’s start, did…

AMT-130, uniQure’s investigational gene therapy for Huntington’s disease, was generally well-tolerated and led to reductions in the levels of mutant huntingtin protein a year after treatment, according to an update from an ongoing Phase 1/2 trial. Moreover, levels of neurofilament light (NfL), a biomarker of nerve damage —…

The first two dosing groups in a Phase 1/2 trial of AMT-130, uniQure’s investigational gene therapy for Huntington’s disease, are now fully enrolled. The trial (NCT04120493), being conducted across 12 U.S. locations, is assessing the safety and tolerability of a single AMT-130 infusion into the brains of…