News

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,…

Huntington’s disease affects an average of 1 in 10,000 people worldwide. But in small, remote pockets of South America, its prevalence skyrockets to nearly 1 in 10. Reasons identified for this include geographic isolation, marriage between cousins, high reproduction rates, and a lack of education.

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

Treatment in a U.S.-based Phase 1/2 clinical trial evaluating uniQure‘s gene therapy AMT-130 in people with Huntington’s disease has been temporarily postponed in response to the COVID-19 outbreak, the company announced. The decision to postpone procedures in the two enrolled…

The uncontrolled jerking and writhing movements associated with Huntington’s disease are well known, along with cognitive decline. But doctors who treat Huntington’s patients are far less familiar with the pain and depression their patients endure almost constantly. That’s the consensus of a panel of experts who spoke at…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…

As a high-school student in the suburbs of Boston, Seth Rotberg watched his mother’s mental and physical health deteriorate because of Huntington’s disease. Rotberg was embarrassed to go out in public. People would stare, despite the 15-year-old’s best attempts to explain that her drunk-like movements were…

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…

In a proof-of-principle experiment, a new gene therapy approach with the potential to treat people with Huntington’s disease has been shown to effectively convert astrocyte brain cells of mice into functioning neurons. Those neurons took the place of nerve cells in the mice that were damaged by…