Alice Melão,  —

Tiny fatty (lipid) particles can be used to enhance the delivery of gene editing tools, such as CRISPR-Cas9, to targeted cells and considerably improve their ability to possibly treat human disorders like Huntington’s disease, researchers reported. Their study, “Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by…

Treatment with EIP Pharma’s investigational therapy neflamapimod (VX-745) can restore nerve cells’ communications and prevent their progressive degeneration and death, new preclinical results show. All together these data further support neflamapimod’s use to treat several neurodegenerative disorders characterized by impairment in nerve…

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…

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…

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…

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…

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…

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…

tMMJ-002, a cannabis investigational product for the treatment of Huntington’s disease, has been granted Orphan Drug designation by the U.S. Food and Drug Administration, announced its developer MMJ International Holdings. This new status is expected to expedite the clinical development, regulatory review, and potential marketing approval of the therapy, which…

A Phase 3 clinical trial is enrolling adults with Huntington’s disease across the United States and Canada to test the potential of the investigational therapy RG6042. The GENERATION HD1 study (NCT03761849) will run in 16 U.S. states and five Canadian provinces, and include about 660 people from 25…