A study with a small cohort of Huntington’s disease patients differed from previous research in finding no metabolic alterations associated with disease progression in stage 2/3 patients compared to both premanifest and healthy subjects. The study, titled “A Metabolic Study of Huntington’s Disease,” was published in the PLOS One journal.
Max Planck Institute of Biochemistry researchers have shown that the intracellular location of protein aggregates, long suspected to be involved in the pathogenesis of neurodegenerative diseases like Huntington’s, is extremely important to the survival of nerve cells. While location in the nucleus has little influence on cellular function, protein deposits in the…
Scientists have identified a proteomic pathway, involving the proteins Homer-3 and mTORC1, that is involved in the degeneration of Purkinje cells, a pathogenesis central to Huntington’s disease. The research paper, “Impaired mTORC1-Dependent Expression of Homer-3 Influences SCA1 Pathophysiology,” was published in Neuron. Purkinje cells (PCs) are the principal neurons…
Using a fruit fly model of Huntington’s disease (HD), researchers found that curcumin – a substance in the spice turmeric– can reduce neuronal death and dysfunction and lead to better motor function in flies. The findings – in the report “Curcumin modulates cell death and is protective in…
Ionis Pharmaceuticals, Inc. — formerly Isis Pharmaceuticals — announced the U.S. Food and Drug Administration (FDA) has approved its Orphan Drug Designation application for IONIS-HTTRx as a potential treatment of Huntington’s disease (HD). Ionis’ IONIS-HTTRx is a generation 2.0+ antisense drug developed to treat HD by reducing the production of all forms of the huntingtin…
VistaGen Therapeutics, Inc., a clinical-stage biopharmaceutical company advancing treatments for depression, cancer, and diseases like Huntington’s that involve the central nervous system, announced that it has secured exclusive worldwide commercial rights to three patent-pending stem-cell technologies from the University Health Network (UHN) of Canada. The technologies were discovered and developed at…
Researchers from the University of Nottingham have discovered that a small molecule called nicotinamide mononucleotide (NMN) has a degenerative effect on axons, the thread-like projections involved in neuronal communication. Their study, titled “Wallerian Degeneration Is Executed by an NMN-SARM1-Dependent Late Ca2+ Influx but Only Modestly Influenced by Mitochondria,” was published in…
Neurodegenerative diseases (ND) represent a major and growing healthcare problem, especially in developed nations with aging populations. These disorders affect mostly adults and can last for decades, causing long-term suffering to patients and their families. Neurodegeneration also contributes to the development of chronic debilitating diseases affecting younger people, such as…
Raptor Pharmaceutical Corp. has announced the efficacy and safety results from CYST-HD, a Phase 2/3 clinical trial conducted in collaboration with the Centre Hospitalier Universitaire d’Angers, evaluating the potential of RP103 as a Huntington’s disease therapy. RP103 is an extended release oral drug candidate currently in clinical development for the treatment of…
A compound tested as a diabetes treatment, KD3010, has shown potential in preclinical tests to reduce Huntington’s disease progression, decrease overall neurodegeneration, and improve motor function. The mouse model study conducted by University of California researchers, “PPAR-δ is repressed in Huntington’s disease, is required for normal neuronal function and can be targeted…
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