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…
News
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…
Oxford Finance, LLC announced that it has finished helping Chosen Healthcare, which operates expert nursing facilities for a variety of illnesses including Huntington’s disease, to recapitalize and position itself for future growth. Chosen Healthcare owns 22 expert nursing facilities and one assisted living facility in Indiana, Iowa and Texas. The organization focus is…
Scientists at the Max Planck Florida Institute for Neuroscience have discovered a crucial switch for understanding how cells in the nervous system communicate. The ground-breaking research could aid in the understanding of nervous system function and also help pinpoint what goes wrong in neurological conditions such as Huntington’s disease. The report, titled “Presynaptic…
Neurodegenerative diseases are caused by excessive neuronal death in distinct brain areas. For example, in Huntington’s disease the striatum is a specifically vulnerable region and disease may benefit from enhanced neuronal protection against oxidative stress. Glyceraldehyde 3-Phosphate Dehydrogenase, known as GAPDH, is a central enzyme in glucose metabolism, but it…
Cholesterol supply to neurons by nanoparticles can improve neuronal and synaptic dysfunction in Huntington’s disease (HD), providing the missing link between the reduction in brain cholesterol observed in HD mice brains and some of the neuronal abnormalities associated with the disease. These are the results of a study entitled “Cholesterol‐loaded…
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