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Sarah Lynn Mays learned she wouldn’t be returning to Northwestern University for her scheduled intrathecal injection of tominersen when her younger sister called to give the news — Roche was stopping dosing for its Phase 3 clinical trial of the Huntington’s disease treatment.
Treatment with tominersen failed to show evidence of clinical benefit in adults with Huntington’s disease during the Phase 3 clinical trial GENERATION HD1. Roche, the company developing tominersen, decided to pause dosing in the clinical trial about a month ago, after a pre-planned review of trial data…
Activities are underway worldwide to mark Huntington’s Disease Awareness Month, set aside each year to call attention to the neurodegenerative disorder that is thought to affect three to seven per every 100,000 people of European ancestry. May 15 is International Huntington’s Disease Awareness Day. Patients, caregivers, family, and friends…
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…
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