News

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…

uniQure is launching a Phase 1/2 trial in the United States to evaluate the safety and effectiveness of its Huntington’s disease investigational gene therapy AMT-130. This follows recent approval of an investigational new drug (IND) application for AMT-130 by the U.S. Food and Drug Administration (FDA). “The…

The progressive death of brain nerve cells observed in Huntington’s disease is likely preceded by the retraction of their neurites — long projections that establish connections throughout the brain, a mouse study reveals. Understanding this mechanism, which is exacerbated during aging, stress, and neurodegenerative diseases, may open new avenues…

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…

The loss of a type of nerve cell called a Purkinje cell in a brain region known as the neocerebellum is associated with the presentation of motor symptoms in Huntington’s disease, a study finds. The study, “Cerebellar degeneration correlates with motor symptoms in Huntington’s disease,” was published…

Asuragen has launched a fast, easy-to-use and reliable diagnostic test, called AmplideX PCR/CE HTT Kit, for Huntington’s disease. The AmplideX test is able to detect up to more than 200 CAG trinucleotide repeats within the gene that codes for the huntingtin (HTT) protein. “AmplideX technology continues to demonstrate…

A method called quantitative electroencephalography (qEEG) enables the identification of Huntington’s gene carriers and could become a disease biomarker, according to a pilot study. The research, “EEG may serve as a biomarker in Huntington’s disease using machine learning automatic classification,” appeared in the journal…

While transplanted fetal nerve cells can survive long term in the brain of a Huntington’s disease (HD) patient, only a specific subset called interneurons thrive, according to a study. Projection neurons — a particular type of nerve cell commonly lost in Huntington’s — were rarely detected, probably due to…

Although well-tolerated, the investigational treatment Huntexil (pridopidine) failed to improve motor functions in a year-long Phase 2 clinical trial of Huntington’s patients. The research, “Safety and efficacy of pridopidine in patients with Huntington’s disease (PRIDE-HD): a phase 2, randomised, placebo-controlled, multicentre, dose-ranging study,” appeared in…

Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…