News

RG6042, formerly called IONIS-HTTRx, a potential therapy for Huntington’s disease, was able to reduce the levels of mutant huntingtin (mHTT) protein in early-stage patients, according to results from a Phase 1/2 clinical trial. Trial results were published in the study, “Targeting Huntingtin Expression in Patients with…

In a newly published set of guidelines, experts give suggestions for the management of some of the behavioral symptoms of Huntington’s disease. By making these guidelines available to both specialists and non-specialists, the hope is that better care and support can be given more consistently to Huntington’s patients and their…

Narrative interventions can help provide support to individuals who are carriers of HTT gene mutations linked to Huntington’s disease, a study shows. The study, “Improving follow up after predictive testing in Huntington’s disease: evaluating a genetic counselling narrative group session,” was published in the…

A newly developed smartphone application, called the Neural Impairment Test Suite app, can run a series of tests and calculate the probability that a user is displaying early symptoms of Huntington’s disease. Developed by a group of researchers at the Kaunas University of Technology (KTU) in…

RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…

Exercise training may help improve cardiovascular and mitochondrial function in patients with Huntington’s disease, with little to no treatment-related adverse events, a review study suggests. The study, “Exercise in Huntington’s Disease: Current State and Clinical Significance,” was published in the journal Tremor and Other Hyperkinetic Movements. Huntington’s…

With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies (NCATS) at…

Experimental immunotherapy laquinimod reduced brain atrophy in the caudate nucleus, one of the most affected brain areas in Huntington’s disease, and other cerebral regions but it did not improve motor function in recently diagnosed patients, LEGATO-HD study findings show. Results from the trial were recently discussed at the…

Mild genetic or environmental changes that upset the circadian clock (our internal sleep-wake cycle) may work to diminish the toxicity of mutant huntingtin, the protein involved in Huntington’s disease and nerve cell death, an early study in a fruit fly model found. The study, “Circadian Clocks Function in Concert…

A protein that can specifically bind to mutated forms of huntingtin and prevent its production — while preserving the normal versions of this protein — may represent a new strategy to attack Huntington’s disease. Researchers from Sangamo and Shire are going to present results from preclinical studies of…