News

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…

Researchers have found a protein, called UBR5, that may reduce neurodegeneration in Huntington’s disease (HD). This protein prevents the toxic aggregation (clustering) of mutant huntingtin protein, which is the underlying mechanism that triggers nerve cells’ death in Huntington’s patients. The study, “The ubiquitin ligase UBR5 suppresses proteostasis collapse in pluripotent…

The level of damage present in the DNA inside the cell nucleus — called nuclear DNA — correlates with disease duration and functional capacity in patients with Huntington’s disease, suggesting its potential use as a disease biomarker, a new study shows. The study, “Increased nuclear DNA damage precedes mitochondrial dysfunction in…

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.

Targeting specific genes involved in inflammation and cellular structure may be a therapeutic strategy to reduce protein clumps in patients with Huntington’s disease, according to researchers. Their study, “High-Throughput Functional Analysis Distinguishes Pathogenic, Nonpathogenic, and Compensatory Transcriptional Changes in Neurodegeneration,” appeared in the journal Cell…

Retrotope’s investigative therapy RT001 reduced cognition deficits in  mice with Huntington’s disease, and was found to lower the amount of oxidative damage in their brains. The study, “Deuterium‐reinforced linoleic acid lowers lipid peroxidation and mitigates cognitive impairment in the Q140 knock in mouse model of Huntington’s disease,” was published in…

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…

Skyhawk Therapeutics and Celgene have entered into a five-year global partnership to use Skyhawk’s STAR* technology platform to discover, advance, and commercialize new small-molecule therapies to treat patients with neurological diseases, including Huntington’s disease. The STAR* approach, which stands for “small-molecule therapies for alternative splicing in RNA,” aims to correct the…

The Scottish region of Fife has launched one of the world’s first local care frameworks for Huntington’s disease, putting local staff and families at the forefront of work to help those with this neurodegenerative disease. The launch, at Victoria Hospital, saw families affected by Huntington’s join health and social…

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.