News

The U.S. Food and Drug Administration (FDA) has granted Voyager Therapeutics permission to start a clinical trial testing VY-HTT01, its investigational gene therapy for Huntington’s disease. Voyager is planning to launch the Phase 1/2 clinical trial, called VYTAL, later this year. VYTAL will be a dose-escalation study, meaning that…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

A chaperone protein called DnaJB8 has an internal molecular “switch” that likely regulates how cells dispose of protein aggregates, like those that cause Huntington’s disease. These findings were detailed in the study “Regulatory inter-domain interactions influence Hsp70 recruitment to the DnaJB8 chaperone,” published in Nature Communications. Huntington’s is…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

A new international consortium, Stem Cells for Huntington’s Disease (SC4HD), has been formed to help facilitate the development of stem cell-based therapies to treat the disorder. The consortium was presented in the Journal of Huntington’s Disease, in the paper, “Stem Cells for Huntington’s Disease (SC4HD): An International…

Anima Biotech has partnered with Takeda Pharmaceuticals to develop small molecules that control mRNA translation — the process by which the information encoded in a person’s DNA is “translated” into a protein — as potential therapies for neurological disorders with a genetic cause, including Huntington’s disease. Huntington’s…

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…

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…

Stalled protein-building may underlie Huntington’s disease, according to data from a recent study. The findings showed that the mutated huntingtin protein, which is at the heart of the disorder, slows down ribosomes, the small cell machinery structures responsible for building new proteins. According to investigators, knowing this and understanding…

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…