The Hereditary Disease Foundation (HDF) raised $900,000 to fund research into Huntington’s disease and other brain disorders during its Nov. 6 “Celebration of 50 Years of Discovery” symposium and gala at New York’s Metropolitan Club. The event included a scientific symposium and a dinner and award ceremony, where winners…
A protein responsible for protecting nerve cells can become embedded in other substances in the brains of Huntington’s patients, preventing it from performing its neuroprotective work, Spanish researchers report. Another important finding was that the areas where the ATF5 protein are embedded also contain clusters of the faulty huntingtin protein…
The U.S. Patent and Trademark Office (USPTO) recently notified Mitochon Pharmaceuticals of its allowance on the first patent that the company says will help it proceed with the continued clinical development of MP101 as a treatment for Huntington’s disease (HD) and others. MP101 is one of…
The Huntington Society of Canada (HSC) and Ottawa-based social media company Shared have jointly launched the “One Extra Push” campaign to raise awareness about Huntington’s disease (HD) in Canada. Shared took up the challenge in honor of Amanda Munro, one of its employees, whose mother has HD. One Extra…
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.
Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age. The effort…
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…
A Spanish study investigating the functioning of chloroplasts — the nutrient-producing “factory” of plants — has revealed a new mechanism that helps understand how a cell controls and regulates protein folding mechanisms. This new finding can improve knowledge of Huntington’s disease and other neurodegenerative disorders such as Alzheimer’s and Parkinson’s…
uniQure recently showed that its gene therapy candidate AMT-130 improves motor coordination and survival in mouse models of Huntington’s disease (HD). The preclinical study results were presented at the European Society of Gene and Cell Therapy (ESGCT) 25th Anniversary Congress, held Oct. 17-20 in Berlin, Germany. The…
More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…
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