News

Prilenia Therapeutics has enrolled more than 120 patients in PROOF-HD, a Phase 3 clinical trial investigating the efficacy and safety of oral pridopidine in early stage Huntington’s disease. That amounts to 25% of the target enrollment of up to 480 patients, the company announced. The trial is…

uniQure has completed patient enrollment for the first group participating in a Phase 1/2 trial assessing the safety and efficacy of AMT-130, its experimental gene therapy for Huntington’s disease…

U.S. lawmakers have again introduced legislation that would speed up the process for Huntington’s disease (HD) patients to get Medicare and Social Security Disability Insurance (SSDI) benefits. The Huntington’s Disease Disability Insurance Access Act of 2021 — S 868/ HR 2050 — would waive the mandatory two-year…

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…