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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…

Researchers have come up with a new method to study the interactions between cell components that are thought to be defective in patients with Huntington’s disease and other neurodegenerative conditions. But while the study answered some initial questions of how these cell components, or organelles, collaborate, much work remains,…

As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…

Three Huntington’s disease (HD) patient advocacy organizations in Europe and North America recently launched a collaborative effort to give patients and the HD community more of a voice in clinical research. The newly formed Huntington’s Disease Coalition for Patient Engagement (HD-COPE) aims to give Huntington’s patients a platform to express their…

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…