News

Researchers have developed a modified and harmless adeno-associated virus (AAV), called AAV-DB-3, that can efficiently deliver low doses of gene therapies to deep regions of the brain impacted by neurodegenerative diseases such as Huntington’s disease. AAV-DB-3 was more than 200 times more potent at delivering its genetic cargo to…

People with Huntington’s disease have greater variations in disease symptoms and physical function between days than within each day, according to a study based on patient reports and objective data collected with a wrist-worn device in the U.S. The findings suggest “it may be sufficient and reliable to evaluate…

Two signaling enzymes, GSK3-beta and ERK1, play opposing roles in the development of Huntington’s disease and may represent new therapeutic targets, a study showed. “We propose that ERK1 may protect neurons in the face of Huntington’s disease, while [GSK3-beta] may exacerbate Huntington’s disease,” Shermali Gunawardena, PhD, the study’s senior…

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, sometimes known as HD, 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…