News

One year of treatment with PTC518 safely lowered blood levels of huntingtin, the protein that’s faulty in Huntington’s disease, in a dose-dependent manner in people with early- and late-intermediate stages of the neurodegenerative condition. That’s according to new data from the Phase 2 PIVOT-HD clinical trial (NCT05358717), which…

The emphasis of this year’s Huntington’s Disease Awareness Month, celebrated each May, is on the patient journey. Also, International Huntington’s Disease Awareness Day is May 15. Huntington’s disease, or HD for short, is a progressive neurodegenerative condition believed to impact 4.9 per 100,000 people globally, including some…

The U.S. Food and Drug Administration (FDA) has designated uniQure’s gene therapy candidate AMT-130 a breakthrough therapy for its potential to slow the progression of Huntington’s disease, according to the developer. This status is intended to speed the development and review of medications for serious or life-threatening illnesses…

Most adults with the genetic defect associated with Huntington’s disease were completely able to perform daily activities and tasks at a first assessment, and maintained such skills over time, according to data from a natural history study of the neurodegenerative condition. That study assessed participants using the total functioning…

Researchers were able to promote the formation of new neurons that integrated into existing motor circuits and helped repair damage and influence motor behavior in the brain of a mouse model of Huntington’s disease, a study showed. “Our research shows that we can encourage the brain’s own cells to…

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…

Daily treatment with Ingrezza (valbenazine) eases the jerky, involuntary movements known as chorea across different body regions in people with Huntington’s disease, according to new data analyses from the KINECT-HD trial. The primary analyses from the Phase 3 KINECT-HD study (NCT04102579) showed that the oral therapy…

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…

One-year results from the Phase 2 PIVOT-HD clinical trial testing PTC Therapeutics’ Huntington’s disease treatment PTC518 are expected in the second quarter. That’s according to an update from PTC, which also noted that in a December meeting with the U.S. Food and Drug Administration (FDA), the agency…

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…