News

Multiple government agencies have cooperated to allow MMJ International Holdings to import cannabis extracts to the United States, for use in potential therapies the company is developing for clinical trials in Huntington’s disease and multiple sclerosis. The cannabis plant makes numerous compounds called cannabinoids, two of the…

Because of the COVID-19 pandemic, Huntington’s Disease Awareness Month organizers have come up with creative ways to show support and call attention to the disorder during May. In past years, May has been full of fundraising and educational activities such as outdoor bake sales, group walks and runs, golf…

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…

AskBio announced that it has acquired BrainVectis, a company developing gene therapies for Huntington’s disease and other neurodegenerative conditions. The two companies will work to advance BrainVectis’ gene therapy programs for neurodegenerative diseases associated with problematic fat metabolism in the brain, including a lead candidate therapy for Huntington’s called…

FFF Enterprises and Bionews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and continue to manage their health during this time when many are finding themselves alone. Recognized as the nation’s leading supplier of…

Roche has completed patient enrollment for its Phase 3 GENERATION HD1 clinical trial evaluating the potential of tominersen to delay the progression of Huntington’s disease. A total of 791 adult patients across approximately 100 clinical sites around the world have been enrolled and the trial is expected to be…

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their homes, one may be a new appreciation of telemedicine. “If something good could come out of this crisis, it’s that…

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…

Vaccinex’s experimental therapy pepinemab (VX15/2503) improves brain metabolism, halts its atrophy, and eases motor and cognitive symptoms in people with late prodromal (before clinical diagnosis) and early Huntington’s disease, according to initial data from a Phase 2 clinical trial. The findings were presented at the Advances…

A DNA-binding compound can reverse the repeat expansion mutation that is associated with Huntington’s disease, an early study reported. This newly developed small molecule was also seen to reduce disease-causing protein aggregates in nerve cells within the brains of mice in a disease model,…