Several small molecules have been identified by the Alborada Drug Discovery Institute as potential inhibitors to help halt the underlying mechanisms that drive the development of neurodegenerative diseases such as Huntington’s and Parkinson’s disease. Silencing phosphatidylinositol 5-phosphate 4 (PI5P4) kinases has been shown to enhance the natural cleaning system of cells, called autophagy,…
Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…
Dysfunction in the development and maturation of the brain’s primary support cells, know as glial cells, contributes to nerve cell damage in a a mouse model of Huntington’s disease. “These new findings help pinpoint how the genetic flaw in Huntington’s gives rise to glial cell dysfunction, which impairs the development and…
The upcoming Phase 3 trial of RG6042 will test whether lowering the levels of a mutant form of huntingtin (mHTT) translates into functional, motor and cognitive benefits for people with Huntington’s disease. Potential benefits could also help advance gene-silencing approaches in other neurodegenerative diseases. “Deep down, we all…
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…
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