News

Efforts to understand brain development in young people at risk for Huntington’s disease have been given a boost by an $18 million grant awarded by the National Institute of Neurological Disorders and Stroke, a division of the…

Huntington’s patients who were treated with the compound WVE-120102 showed a statistically significant drop in mutant huntingtin protein (HTT) in their cerebrospinal fluid, according to a recent Phase 1b/2a clinical trial. The compound was also found to be safe and well-tolerated. These data support the testing of higher dosages…

Certain aspects of Huntington’s disease (HD), such as the biologic mechanisms of depression, are not consistently replicated in mouse models of the disorder, which may hinder therapy development, a study reports. The study, “Increased Serotonin Transporter Expression in Huntington’s Disease Patients Is Not Consistently Replicated in Murine Models,”…

Abnormal development of neural tissues may precede neurodegeneration in early-onset juvenile forms of Huntington’s disease, a recent study has found. Researchers also showed how targeting the cellular pathway involved in this process might be an effective strategy to restore normal neural development. Their study, “Expanded huntingtin CAG…

A small RNA molecule known as microRNA-1, found in both worm and human cells, can promote the removal of toxic protein aggregates — hallmarks of neurodegenerative diseases — by triggering a self-cleaning process called autophagy, a study shows. These findings may open new avenues for potential therapeutics for disorders such…

Joining a “co-produced” walking group can be beneficial for people with Huntington’s disease, increasing their confidence, strength, and ability to walk, a study found. However, further research is needed to find ways to motivate Huntington’s patients to join such programs — and to keep the groups going, the investigators…

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…

An inability to correctly identify face-like objects and fewer brain signals associated with facial recognition were found in people with Huntington’s disease who had yet to show symptoms, suggesting these tests can be a marker of early disease progression, a study reports. …

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.

Researchers have identified three new small molecules that significantly reduce the formation of toxic aggregates of mutant huntingtin — the underlying cause of Huntington’s disease — in cells derived from Huntington’s patients. These molecules work by selectively and potently binding to the expanded CAG repeats in an intermediate molecule…