The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…
When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience. Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…
Administration of the hormone erythropoietin (EPO) using a gene transfer method improved spatial cognition, stimulated the branching of nerve stem cells, and preserved the brain region known as the hippocampus in a mouse model of…
Vico Therapeutics has raised $31 million in Series A financing to continue pursuing the development of RNA modulating technologies that could potentially act as treatments for central nervous system disorders. It is the company’s first significant round of investment funding. At the forefront of Vico’s plans is further…
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…
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