News

Huntington’s disease alters nerve cell development in the cortex — a brain region largely affected in people with the condition — before birth, a study has found. “Huntington’s definitely has a neurodevelopmental component in addition to a neurodegenerative disease,” Sandrine Humbert, PhD, the study’s senior author at the…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

Microbes in the gut of people with Huntington’s or likely to develop this disease appear to differ from healthy peers, opening the door to potential new biomarkers and therapeutic targets, according to a recent study. The study, “Gut dysbiosis in Huntington’s disease: associations between gut microbiota, cognitive…

Antidopaminergic medications are associated with lesser chorea and irritability in people with Huntington’s disease, but they may worsen their cognitive skills, data from the largest observational study of the disease show. Future, prospective studies are needed to confirm these findings, and to determine whether treatments to lower dopamine levels indeed…

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…