Patricia Inácio, PhD, science writer —

Nerve cells contain proteins that send signals to other cells and proteins that receive neighboring cells’ signals. Researchers at Rensselaer Polytechnic Institute in Troy, New York, are trying to understand how the sending proteins end up on one side of a nerve cell’s membrane and the receiving proteins on the…

uniQure expects to advance the clinical development of its Huntington’s disease investigational gene therapy, called AMT-130, during 2018. The company says it plans to file an investigational new drug application (IND) with the U.S. Food and Drug Administration (FDA) in the second half of 2018. The first human clinical trial…

Aberrant metabolism of urea is a potential first event promoting Huntington’s disease (HD), a study with a sheep model and human brain samples showed. The findings suggest that lowering brain levels of urea is a potential therapeutic avenue for HD. The report “Brain urea increase is an early Huntington’s…

Researchers have found that rilmenidine, a blood pressure medicine, was relatively safe and well-tolerated by Huntington’s disease (HD) patients in a small study. However, its effectiveness in treating the disease remains unknown and warrants further investigation. The study, “An open-label study to assess the feasibility and tolerability of rilmenidine for…

Researchers have identified an elaborate protein quality control system that prevents toxic accumulation of faulty proteins inside mitochondria, a phenomenon that underlies many neurodegenerative diseases, including Huntington’s disease. The study, ”Cytosolic Protein Vms1 Links Ribosome Quality Control to Mitochondrial and Cellular Homeostasis,” was published in the journal Cell.

As therapeutics for Huntington’s disease (HD) are still a need, a number of natural compounds may hold promising beneficial effects, a review study shows. The review “The Medicinal Chemistry of Natural and Semi-Synthetic Compounds Against Parkinson’s and Huntington’s Diseases” was published in the journal ACS Chemical Neuroscience. As…

Huntington’s disease causes hearing problems, a study with a small sample size suggests. The research, “Auditory dysfunction in patients with Huntington’s disease,“ was published in Clinical Neurophysiology. A severe, inherited neurodegenerative disease, Huntington’s is characterized by movement problems, cognitive deterioration and behavioral disorders. Many people with other neurodegenerative…

Researchers generated the first human line of Huntington’s disease induced pluripotent stem cells (iPSCs) that do not express the dysfunctional protein underlying the condition. The cells represent a potential resource to investigate the function of the huntingtin protein in vitro (in a lab culture dish or test tube). The study,…

Researchers used gene editing to suppress the activity of protein mutations in the brains of mice with  Huntington’s disease. Suppressing the huntingtin protein alleviated early signs of the disease and reduced the mice’s movement problems, the study reported. The research, “CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of…

Probiodrug AG recently announced the results of a preclinical study investigating the role of glutaminyl cyclases (QCs) in Huntington’s disease. The results will be presented at the 12th Annual HD Therapeutics Conference of the CHDI Foundation, in Malta, April 24-27. Probiodrug AG is a biopharmaceutical company developing innovative…