Prilenia Therapeutics, in partnership with the Huntington Study Group (HSG), has launched the Phase 3 PROOF-HD clinical trial to evaluate the company’s lead experimental oral therapy, pridopidine, in people with early stage Huntington’s disease. Pridopidine “is a first-in-class [therapy] candidate, with promising previous clinical results and…
A new book is available to help children cope when a family member has Huntington’s disease. Called “Harry needs a hug: A story for children learning to deal with Huntington’s Disease,” the 32-page book was written by Emma Terranova, founder of Campaign For My Brain, a United…
Behavioral changes due to early onset Huntington’s disease and problems with school, and social and family life are common, particularly among pediatric patients, according to a case series study in Germany. Socio-medical problems in these children were associated both with their own disease, and with the additional burden of parents…
An enzyme called TANK‐binding kinase 1 (TBK1) can lower levels of the mutant huntingtin protein — which underlies the development of Huntington’s disease — and prevent its aggregation in cellular and animal models of the disease, a study reports. Data showed that TBK1 is able to do this by…
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 —…
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