News

The Huntington’s Disease Digital Motor Score (HDDMS), a new measure based on four motor tests that are done through a smartphone app, may be able to detect Huntington’s disease progression with greater sensitivity than current standard tools, a study reports. “Our findings suggest that incorporating the HDDMS in clinical trials…

Introducing small interruptions into the abnormally long CAG repeat expansion that causes Huntington’s disease can stabilize or even reduce the length of that DNA stretch, which would be expected to slow disease progression. These are the findings of a study that tested base editing, a technique that changes a…

uniQure plans to submit a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) early next year seeking approval of its gene therapy candidate AMT-130 for Huntington’s disease. Top-line, three-year data from the ongoing clinical trials that will support that application are due in the…

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…