News

Active psychiatric disorders are associated with increased risk of suicidal behaviors among Huntington’s patients participating in clinical trials, a large-scale study suggests. The study, “Risk factors for suicidality in Huntington disease: An analysis of the 2CARE clinical trial,” was published in Neurology. Huntington’s disease is mostly recognized for…

Practicing contemporary dance can improve motor function in people with Huntington’s disease, a new study has found. Titled “Contemporary Dance Practice Improves Motor Function and Body Representation in Huntington’s Disease: A Pilot Study,” the study was published in the Journal of Huntington’s Disease. There is overwhelming evidence…

A software known as “DriveSafe DriveAware” can help evaluate if patients with Huntington’s disease still have the cognitive fitness needed to safely drive a car, a pilot study shows. However, to increase the accuracy of the software some improvements should be made to better fit the profile of patients…

The U.S. Food and Drug Administration (FDA) has issued a letter of support encouraging sRNAlytics to further develop its sRNA-FIND platform for discovering and monitoring variants of small RNA molecules that may work as biomarkers for tracking progression of Huntington’s disease decades before symptom onset. In addition, these biomarkers may…

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bathdissertation, she analyzed Brexit’s long-term impact…

Analysis of behavior patterns in genetically engineered mini pigs could be a valuable tool for assessing Huntington’s disease symptoms, a study shows. This new animal model may help researchers further explore the progression of the disease, as well as daily symptom fluctuations and new strategies to manage them. The…

The U.S. Food and Drug Administration has granted orphan drug designation to MP101, an investigational mitochondrial-targeted neuroprotective agent, to treat Huntington’s disease. Mitochon Pharmaceuticals, the therapy’s developer, expects to begin a Phase 1 clinical trial in healthy volunteers in 2019 and move to Phase 2 studies in 2020. “We are…

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…

Encouraging new preclinical data on AMT-130, uniQure’s experimental gene therapy candidate for Huntington’s disease, were presented at the 14th Annual Huntington’s disease Therapeutics Conference. The conference was organized by the CHDI Foundation and held in Palm Springs, California, Feb. 25–28. “Findings from our preclinical studies illustrate…

Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those illnesses and highlighting…