Patricia Inácio, PhD,  science writer—

A Phase 1/2 clinical trial testing uniQure’s potential gene therapy AMT-130 in people with Huntington’s disease can continue enrollment, according to a positive review by the study’s independent data safety monitoring board (DSMB). The DSMB reviewed safety data from four patients given the higher dose of the therapy, which…

A Phase 3 clinical trial investigating the efficacy and safety of oral pridopidine for those with early stage Huntington’s disease is now underway at more than 50 clinical sites in the U.S., the U.K., Canada, and Europe, according to Prilenia Therapeutics, which is developing the treatment candidate. Prilenia…

The U.S. Food and Drug Administration (FDA) has granted an orphan drug designation to SOM3355, an investigational treatment for chorea, or involuntary jerky movements, associated with Huntington’s disease, its developer SOM Biotech has announced. Orphan drug status helps to encourage the development of therapies for rare…

A Phase 1/2 trial evaluating Wave Life Science’s investigational therapy WVE-003 for Huntington’s disease has started dosing patients, the company has announced. The SELECT-HD trial (NCT05032196), underway in clinical sites in Australia, Germany, Poland, and the U.K., is currently looking to enroll 36 Huntington’s patients, ages…

A history of high alcohol intake seems to be linked to increased motor impairment and a higher burden of psychiatric symptoms in Huntington’s disease patients, a large international study suggests. The observational study, “Genetic and Environmental Contributors to Neurodegeneration: An Exploration of the Effects of Alcohol…

Treatment with pridopidine rescues the function of mitochondria — the cell’s powerhouses — boosting energy production, and reduces oxidative stress in models of Huntington’s disease, preclinical research shows. According to researchers, these findings support the therapeutic potential of pridopidine and its ongoing clinical development. Pridopidine is a small oral…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

A Phase 1/2 trial testing the safety and efficacy of AMT-130, uniQure’s experimental gene therapy for Huntington’s disease, can continue dosing after a positive recommendation from an independent board. The data safety monitoring…

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…

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…