News

Raman spectroscopy, an analysis technique that uses light, identified a specific molecular fingerprint in the blood of people with Huntington’s disease, a study has found. This technique may be useful as a non-invasive diagnostic tool to track disease progression and as…

Railway signalman Sandy Patience has witnessed the devastating effects of Huntington’s disease on family members. When he learned two years ago that he, too, inherited the neurodegenerative disorder, the news was crushing. But now, as one of the first Scotland residents to participate in an international Phase 3 trial…

As many as 80 experts from a dozen countries will gather next month in Vienna for the European Conference on Controversies in Huntington’s Disease (ECCH2020). The Feb. 13–14 meeting “aims to increase awareness and interests around the main aspects” of Huntington’s disease, which according to the European…

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…