News

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…

Enroll-HD, the world’s largest observational study of Huntington’s disease, hopes to add thousands of patients to its registry, reaching a possible total of 30,000 over the next three years. Georg Bernhard Landwehrmeyer, MD, its principal investigator, spoke about the ambitious project at last month’s European Conference on Controversies…

A Phase 1 clinical trial testing Roche’s investigational therapy tominersen for Huntington’s disease has been placed on hold after two cases of infection were found during the study. The infections were linked to the device used to take samples of patients’ cerebrospinal fluid (CSF, the…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…