News

AMT-130, uniQure’s investigational gene therapy for Huntington’s disease, was generally well-tolerated and led to reductions in the levels of mutant huntingtin protein a year after treatment, according to an update from an ongoing Phase 1/2 trial. Moreover, levels of neurofilament light (NfL), a biomarker of nerve damage —…

Scientists have created a map of the cells contained in human brain blood vessels. Using the map as a reference, they have discovered certain gene changes occurring in specific brain cells in Huntington’s disease. The study, “Single-cell dissection of the human brain vasculature,” was published in Nature.

Bit.Bio has launched a new human cell model to advance research and accelerate therapeutic development for Huntington’s disease. The cells, called ioGlutamatergic Neurons HTT-50CAG/T, are engineered to carry a specific mutation in the HTT gene that causes Huntington’s. By carrying the same genetic…

The National Organization for Rare Disorders’ (NORD) “Living Rare, Living Stronger Patient and Family Forum” is back in person on June 26 for a day of learning and networking in Cleveland, Ohio. The event, which brings together the rare disease community, will take place at the InterContinental Cleveland Conference…

Activating the JAK2-STAT3 pathway in astrocyte cells reduced multiple hallmark features of Huntington’s disease  — including the toxic clumping of the huntingtin protein in nerve cells — from the brains of a mouse model, a study reported. “Our results open new therapeutic avenues to further enhance the natural partnership…

The U.S. Food and Drug Administration (FDA) has designated valbenazine an orphan drug as a potential treatment to control the involuntary, jerky movements known as chorea in people with Huntington’s disease. Orphan drug status is given to therapies with the potential to improve medical care for rare disease. It…

Clumps of protein resulting from HTT mutations, the underlying cause of Huntington’s disease, form distinct structures in different parts of the cell, according to detailed imaging analysis. The findings suggest these clumps form by different mechanisms and may require different therapeutic strategies to block their formation and toxicity that…

Some 41,000 U.S. residents are thought to live with Huntington’s disease (HD), a rare progressive disorder that breaks down brain nerve cells. During May, and as part of Huntington’s Disease Awareness Month, the Huntington’s Disease Society of America (HDSA) is launching a robust social media initiative aimed at…

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult,…

The experimental therapy pepinemab can aid cognition in people with early Huntington’s disease who showed signs of cognitive impairment when they started treatment, new analyses from the Phase 2 SIGNAL clinical trial indicate. Findings were presented at the virtual American Academy of Neurology 2022 Annual Meeting in the poster, “…