Alice Melão,  —

Vaccinex has completed its target enrollment in a Phase 2 clinical trial assessing the potential of the investigational antibody VX15/2503 (pepinemab) as a treatment for patients with early manifest and late prodromal (before clinical diagnosis) Huntington’s disease. The ongoing trial (NCT02481674), known as SIGNAL, is the first to investigate a…

One-time delivery of Voyager Therapeutics’ lead candidate gene therapy, VY-HTT01, effectively reduced the levels of the faulty gene responsible for Huntington’s disease in important brain areas of non-human large primates. These positive preclinical results, together with other ongoing studies, are expected to support the submission of an investigational new…

The National Institute on Aging at the National Institutes of Health (NIH) has granted $12.6 million to Northwestern University to study the role of protein quality control mechanisms in human aging and neurodegenerative diseases, including Huntington’s. The five-year grant is one of the NIH’s Program Project Grants, designed to assemble…

Short huntingtin (HTT) protein fibers precede the formation of larger deposits that accumulate in the brains of those affected by Huntington’s disease, preclinical results reveal. These findings one day may help researchers predict the onset and course of the disease. The study, “mHTT Seeding Activity: A Marker of…

The European Medicines Agency (EMA) has granted Priority Medicine (PRIME) designation to Roche’s investigational therapy RG6042 for the treatment of Huntington’s disease. PRIME provides a pathway for expedited assessment and review by the EMA, potentially enabling the drug to reach patients sooner. “We are very pleased…

SOM3355 — a medicine approved for high blood pressure and chest pain — is being tested in a Phase 2 trial as a means to treat chorea in Huntington’s disease patients, SOM Biotech announced. The proof-of-concept study (NCT03575676) will recruit participants in four clinical sites in or near Barcelona, Spain.

Adequate support and follow-up should be considered when implementing and designing pre-symptomatic genetic testing for Huntington’s disease, a study has revealed. The study, “Perspectives on Genetic Testing and Return of Results from the First Cohort of Presymptomatically Tested Individuals At Risk of Huntington Disease,” was published in…

Improved measures to accurately determine the effectiveness of treatments targeting Huntington’s disease are necessary, according to clinical neurologists and Huntington’s specialists. The editorial, “Improved metrics for Huntington’s disease trials,” reflects on recent advances made for the development of targeted agents that can effectively change the course of Huntington’s disease.

A new therapeutic strategy based on a small part of the huntingtin protein — which in a mutated form is responsible for Huntington’s disease — shows potential to regulate the course of disease, according to a study using mice. The study, “Improvement of BDNF signalling by P42…

Apathy is linked to physical, cognitive, and behavioral impairments in patients with Huntington’s disease, a study shows. The study, “Relationships Among Apathy, Health-Related Quality of Life, and Function in Huntington’s Disease,” was published in The Journal of Neuropsychiatry and Clinical Neurosciences. Huntington’s disease is characterized by…