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The U.S. Food and Drug Administration (FDA) has awarded orphan drug status to uniQure’s proprietary candidate for Huntington’s disease (HD), a first in the history of Huntington’s treatment. No approved therapies exist to target the underlying cause of Huntington’s, a disease caused by a mutation in the huntingtin gene .

Identifying Huntington’s disease progression can be a challenge in clinical trials. Now, an international team of researchers proposes in an article published in JAMA Neurology the use of a new composite measure to help evaluate disease progression prior to a motor diagnosis. Progression of Huntington’s disease is characterized by the…

The National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH) has awarded a grant of more than $2.5 million to support the development of HDWear, a telecare technology that uses wearable sensors to remotely monitor motor function in Huntington’s disease (HD) patients.

Less is known about the late stages of Huntington’s disease than about its earlier stages, particularly concerning patients enrolled in hospice care. Researchers studying this patient group recently concluded that they would benefit from more extensive involvement of hospice in end-of-life care. The study, “Utilization of hospice services in…

Three entities — the Clinical Data Interchange Standards Consortium (CDISC), the Critical Path Institute (C-Path) and the CHDI Foundation (CHDI) — have announced that the Huntington’s disease (HD) Therapeutic Area User Guide (TAUG-HD v1.0) is up for public review through Nov. 10. TAUG-HD v1.0 was developed through the Coalition…

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,…

AC Immune has identified antibodies that target the alpha-synuclein and TDP-43 proteins associated with neurodegenerative diseases such as Huntington’s. The discovery could be used to develop treatments for such diseases, the company said. Antibodies are immune system cells that can recognize and generate a response to invaders. “Many neurodegenerative diseases share…

A new version of the CRISPR-Cas9 gene editing technique designed to target RNA molecules can fix the underlying cause of Huntington’s disease and similar disorders, say researchers at the University of California San Diego. Their study, “Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9,” appeared in…

Researchers in England and Wales have identified a gene linked to disease progression in Huntington’s disease, shedding new light on the poorly understood mechanisms that cause the disorder. Although the results — published in the journal The Lancet Neurology —hold no immediate prospect of leading to a cure, researchers say…

Aggregates of mutant huntingtin protein have been associated with cellular toxicity in Huntington’s disease. Now researchers have discovered that isolated forms of mutated huntingtin are actually responsible for major molecular changes in cells that will drive toxicity. The study, “Transcriptional profiles for distinct aggregation states of mutant Huntingtin exon…