News

People who will develop Huntington’s disease have subtle biological evidence of brain damage decades before the onset of symptoms, a new study indicates. This finding could be important to determine when to begin treatments for Huntington’s. The study, “Biological and clinical characteristics of gene carriers far…

Among people with Huntington’s disease living in India, psychiatric problems are common but routine clinical follow-up is lacking, a study suggests, emphasizing the need for better symptomatic care. The study, “Psychiatric morbidity and poor follow-up underlie suboptimal functional and survival outcomes in Huntington’s disease,” was published in …

The first patients have been enrolled in a clinical study of the natural history of Huntington’s disease (HD) and its biomarkers, to better inform how to measure and monitor early signs of the disease and support future clinical studies. The SHIELD HD study, sponsored and led by Triplet…

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…

AFFiRiS’ C6-17, a candidate monoclonal antibody for the treatment of Huntington’s disease, is able to target and halt the spread of mutant huntingtin protein — the underlying cause of the neurodegenerative disorder — across cells, new preclinical data show. The study, “Inhibiting cellular uptake of mutant huntingtin using…

A Phase 1/2 clinical trial evaluating AMT-130 as a potential gene therapy for Huntington’s disease has “treated” its first two patients, uniQure announced. One person was given AMT-130 and the other a sham surgery as part of a control group. Both will now be followed for three months, after which a Data…

At least three major pharmaceutical companies are looking into potential gene therapies for Huntington’s disease — a rare inherited condition without a cure or a treatment to stop its progression. While several approved therapies exist to control the disorder’s physical and psychiatric effects, non-drug alternatives to alleviate the worst…

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…

Austedo (deutetrabenazine) can be safely taken at higher than the maximum dose now approved to control involuntary movement in people with Huntington’s disease, an analysis of clinical trial data reported. These findings, “Evaluation of the Safety of Deutetrabenazine at Higher Doses to Treat Chorea in…

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.