Marisa Wexler, MS, senior science writer —

Branaplam (LMI070), an experimental oral therapy for Huntington’s disease that’s currently in early clinical trials, reduces levels of the mutant huntingtin protein by interfering with a process called mRNA splicing, a new study reveals. “Here, we delineated the mechanism of action of Branaplam,” researchers wrote. The study, “…

Palliative care training for all clinicians on an interdisciplinary team can increase the number of discussions about advanced care planning for people with Huntington’s disease, a new study shows. “To our knowledge, this is the first description of primary palliative care for Huntington’s disease in which all interdisciplinary team…

Many U.S. clinics treating people with Huntington’s disease reported difficulties in accessing needed professionals — such as genetic counselors, social workers, and dietitians — to help provide multidisciplinary care to patients with the progressive disorder, according to a survey-based study. Researchers billed the study, which involved more than 150…

Reduced volumes of gray matter in certain regions of the brain are tied to poorer quality of life in people with Huntington’s disease, a new study reports. The study, “Brain, cognitive, and physical disability correlates of decreased quality of life in patients with Huntington’s disease,” was…

Due to possible side effects, dosing is being paused in the Phase 2 VIBRANT-HD clinical trial of oral branaplam (LMI070) in adults with Huntington’s disease. A scheduled assessment found “early signs” that “branaplam might be causing peripheral neuropathy,” a condition marked by pain or numbness in the extremities caused by damage…

Long-term trials of interventions to promote physical activity for people with Huntington’s disease are feasible, with a “nested trial” approach that assigns some patients already taking part in an observational study to an activities group while others serve as controls, a study reported. The yearlong effort was designed as a…

Levels of neurofilament light chain, a marker of nerve cell degeneration, are increased in children with juvenile-onset Huntington’s disease (JOHD), according to a new study. Results also suggest that levels of this protein, known as NfL, are correlated in these young patients both with disease severity and damage to…

Most young people at risk of developing Huntington’s disease are not given information about the disorder at the time they’re told about the risk and end up seeking it on their own online, according to a new study. “This study is the first to take an in-depth look at…

The U.S. Food and Drug Administration (FDA) has designated valbenazine an orphan drug as a potential treatment to control the involuntary, jerky movements known as chorea in people with Huntington’s disease. Orphan drug status is given to therapies with the potential to improve medical care for rare disease. It…

The experimental therapy pepinemab can aid cognition in people with early Huntington’s disease who showed signs of cognitive impairment when they started treatment, new analyses from the Phase 2 SIGNAL clinical trial indicate. Findings were presented at the virtual American Academy of Neurology 2022 Annual Meeting in the poster, “…