News

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…

NeuExcell Therapeutics has entered into a research deal with Spark Therapeutics to advance the development of a safe and effective neuroregenerative gene therapy for Huntington’s disease. Under the terms of the agreement, Spark, a member of the Roche Group, will have access to NeuExcell’s gene therapy platform…

A few years ago, Shelby Lentz wrote a song called “Champions,” originally inspired by the “Rocky” movies. The plucky country anthem has since taken on a deeper, more personal meaning as she and members of her family battle Huntington’s disease. “We wrote this entire song, and I recorded…

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…

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to SAGE-718 as a potential therapy for Huntington’s disease. The FDA gives this designation to experimental medications with the potential to improve medical care for serious health conditions and fill unmet needs. The designation gives the therapy’s…

The interaction between FAN1 and MLH1 — two DNA repair proteins known to be genetic modifiers of Huntington’s disease — protects against further expansion of disease-causing CAG repeats, according to a study using human and mouse models of the disease. Specifically, this interaction prevents MLH1’s recruitment to a DNA…

No significant safety concerns were identified in the first eight Huntington’s disease patients treated with uniQure’s potential gene therapy AMT-130 in a U.S. Phase 1/2 clinical trial. Six of these patients received the low dose of the therapy — which is delivered directly into the brain — and two…

Huntington’s disease patients who have not yet developed symptoms have a different relationship with time than otherwise-healthy individuals, according to a new study based on patient interviews. The findings highlight a need for long-term psychological support for people with presymptomatic Huntington’s to help them develop proactive coping strategies and…

Brain cells called glia work to protect the brain from damage in Huntington’s disease by reducing the expression of genes involved in maintaining connections between nerve cells, a new study indicates. The findings suggest that targeting glia could be a useful strategy for Huntington’s treatment, and also highlight that…