The international Huntington’s Disease Regulatory Science Consortium (HD-RSC) developed a biological definition of Huntington’s disease (HD) and a staging system that, for the first time, covers the entire disease course. The development and validation processes of the new framework, called the Huntington’s Disease Integrated Staging System (HD-ISS), were…
Spending more time on leisure activities — which contributes to a person’s cognitive reserve, or the ability to improvise — is associated with lesser cognitive and functional decline in people with early manifest Huntington’s disease, a study shows. In addition, a greater number of CAG repeats within the HTT gene…
Throughout 2021, Huntington’s Disease News brought you daily coverage of the latest scientific findings, treatment developments, and clinical trials related to Huntington’s disease. As a reminder of what mattered most to you last year, here are the top 10 most-read articles of 2021 with a brief description of what made…
The U.S. Food and Drug Administration (FDA) has given fast track designation to Prilenia Therapeutics’ pridopidine as a potential oral treatment for Huntington’s disease. This designation is given to therapies that show considerable potential in addressing serious conditions for which available treatments fall short. It is meant to…
The interaction between FAN1 and MLH1 — two DNA repair proteins known to be genetic modifiers of Huntington’s disease — protects against further expansion of disease-causing CAG repeats, according to a study using human and mouse models of the disease. Specifically, this interaction prevents MLH1’s recruitment to a DNA…
No significant safety concerns were identified in the first eight Huntington’s disease patients treated with uniQure’s potential gene therapy AMT-130 in a U.S. Phase 1/2 clinical trial. Six of these patients received the low dose of the therapy — which is delivered directly into the brain — and two…
Progression in Huntington’s disease may be due more to a gradual loss of the system that maintains the health of brain cells than to accumulated damage caused by the disease itself, a computational analysis of large and complex datasets suggests. This analysis, called Geomic, showed that while brain cells most vulnerable…
Roche has discontinued dosing in the Phase 3 GENERATION HD1 trial of its investigational therapy tominersen in adults with Huntington’s disease. The decision was made after a pre-planned review of trial data by an independent data monitoring committee, which issued a “no go” recommendation based on tominersen’s potential benefit/risk…
Women with Huntington’s disease have worse motor, cognitive, and depressive symptoms than men with the condition, according to data from the global Enroll-HD observational study. Notably, no sex-specific differences were found for age at diagnosis and symptom progression over the four years of follow-up. These findings help to increase…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…
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