Marisa Wexler, MS, senior science writer —

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases.  “The RareLaunch program is…

The U.S. Food and Drug Administration (FDA) has given orphan drug designation to the oral therapy branaplam (LMI070) as a potential treatment for Huntington’s disease, the therapy’s developer, Novartis, announced. The designation is given to medications that have the potential to treat rare diseases — defined in…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

Vaccinex’s investigational antibody pepinemab showed good tolerability in a Phase 2 clinical trial of people with late prodromal (before clinical diagnosis) and early Huntington’s disease (HD), top-line data show. The results also hint the treatment may improve cognition; however, those efficacy results did not reach statistical significance.

People who will develop Huntington’s disease have subtle biological evidence of brain damage decades before the onset of symptoms, a new study indicates. This finding could be important to determine when to begin treatments for Huntington’s. The study, “Biological and clinical characteristics of gene carriers far…

Among people with Huntington’s disease living in India, psychiatric problems are common but routine clinical follow-up is lacking, a study suggests, emphasizing the need for better symptomatic care. The study, “Psychiatric morbidity and poor follow-up underlie suboptimal functional and survival outcomes in Huntington’s disease,” was published in …

Austedo (deutetrabenazine) can be safely taken at higher than the maximum dose now approved to control involuntary movement in people with Huntington’s disease, an analysis of clinical trial data reported. These findings, “Evaluation of the Safety of Deutetrabenazine at Higher Doses to Treat Chorea in…

A novel class of proteins, dubbed heat-resistant obscure (Hero) proteins, can protect other proteins from a variety of cellular stresses, and also can prevent the formation of protein aggregates typical of Huntington’s disease and amyotrophic lateral sclerosis (ALS). The new protein class was described in the study “…

Multiple government agencies have cooperated to allow MMJ International Holdings to import cannabis extracts to the United States, for use in potential therapies the company is developing for clinical trials in Huntington’s disease and multiple sclerosis. The cannabis plant makes numerous compounds called cannabinoids, two of the…

People with Huntington’s disease who develop psychosis tend to have worse cognitive and behavioral disturbances, reduced ability to function, and fewer involuntary movements than patients who do not experience these psychiatric symptoms, a new study reports. The study, titled “Psychosis and longitudinal outcomes in Huntington disease: the…