Alnylam Pharmaceuticals has launched a Phase 1 clinical trial to test the safety and pharmacological properties of its treatment candidate ALN-HTT02 in adults with Huntington’s disease, the company announced as part of a financial update. “We [have] made great strides with our pipeline, … initiating a Phase 1…
Higher levels of an enzyme called GST02 in the most affected neurons in Huntington’s disease could underlie early increases in the brain-signaling chemical dopamine that are believed to drive Huntington’s motor symptoms, according to a mouse study. Scientists had previously uncovered that a deficiency in a signaling pathway called…
Blood levels of neurofilament light protein (NfL) in people who carry Huntington’s disease-causing mutations, but aren’t showing symptoms, can identify those who will develop the disease in the next decade. These are the findings of a 14-year study that also showed that greater increases in blood NfL levels, a…
The U.S. Food and Drug Administration (FDA) is supporting financially a study into how well wearable sensors measure two early signs of Huntington’s disease, which could be of use to clinical trials of potential treatments. The funding comes from the agency’s Digital Health Technologies for Drug Development…
Loqus23 Therapeutics has raised 35 million pounds (about $45 million) in financing to advance its lead oral small molecule candidate for Huntington’s disease through preclinical development and early clinical studies, which are anticipated in 2026. The series A financing round was led by Forbion, a life sciences…
Unlike its healthy counterpart, the mutated version of the huntingtin protein that drives Huntington’s disease may keep nerve cells from repairing their damaged DNA, causing them to die, a study showed. Specifically, the mutated protein failed to stimulate the production of poly ADP-ribose, known as PAR — a molecule…
In Huntington’s disease (HD), progression is associated with metabolite changes in the brain that generally worsen as patients move from a presymptomatic stage to overt symptom onset, a review study reports. Its scientists looked at data from published studies analyzing metabolic changes in the brain between people with Huntington’s…
Vico Therapeutics’ investigational therapy VO659 was found to lower levels of the disease-causing mutant huntingtin (HTT) protein — known as mHTT — in the cerebrospinal fluid (CSF), the liquid that surrounds the brain and spinal cord, of people with Huntington’s disease. Additionally, treatment was not associated with an…
The European Medicines Agency (EMA) has agreed to review Prilenia Therapeutics’ application seeking approval of pridopidine, an oral therapy candidate for adults with Huntington’s disease, the company announced. The regulatory application is based on safety and efficacy data from pridopidine’s clinical development program, which showed the therapy…
Examining the amounts of certain microbes in the gut and levels of specific metabolites in the blood may help distinguish people with and without Huntington’s disease with 100% accuracy, a study suggested. “This study determined crucial functional gut microbiota and potential biomarkers associated with [Huntington’s development], providing new insights…
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