News

The genetic mutation behind Huntington’s disease may cause neurodegeneration through the production a toxic form of the huntingtin protein, but abnormally long RNA molecules that form gel-like structures that gum up protein production might play a role too. These are the findings of a new study that’s part of…

The once-daily oral therapy Ingrezza (valbenazine) — already marketed in the U.S. as a treatment for a movement disorder — has now been approved by the U.S. Food and Drug Administration (FDA) for chorea in adults with Huntington’s disease. Chorea, characterized by uncontrolled, jerky movements that can…

Caregivers of people with Huntington’s disease want support programs that will help them in balancing their own care needs over time with those of the patient, often a family member. This theme emerged from caregiver focus groups conducted in the Netherlands, where support was considered effective if it would…

With early data on AMT-130 for Huntington’s disease reportedly looking promising, uniQure said it’s planning to meet early next year with regulatory authorities to discuss the late-stage development of its gene therapy candidate. By that time, more data on the ongoing Phase 1/2 clinical trials testing the therapy…

Increased levels of the stress response protein MTF1 suppressed the toxic effects of the mutant huntingtin (mHTT) protein — the underlying cause of Huntington’s disease — in cellular and animal models of the neurodegenerative condition, a study showed. Specifically, MTF1 was found to counteract mHTT-associated oxidative stress, a type…

People carrying fewer Huntington’s disease-causing CAG repeats in the HTT gene — who traditionally are thought to have milder disease — show similar cognitive deficits to those with more CAG repeats, a study found. However, patients with fewer repeats are less likely to show Huntington’s characteristic involuntary muscle contractions,…

When young, healthy progenitors of neuron-supporting cells called glia are transplanted into the adult brain, they can grow and replace diseased glial cells, including those carrying a Huntington’s disease-causing mutation, a study in mice reports. “These findings have strong therapeutic implications, as they suggest that in the adult human…

Huntington’s disease could develop at earlier ages in people using calcium channel blockers to control hypertension, a study that looked into genes encoding targets of blood pressure-lowering medications reported. Findings may have important implications for managing high blood pressure in people at risk of Huntington’s, identified before disease…

PTC Therapeutics’ Huntington’s disease treatment candidate PTC518 is well-tolerated and lowers blood levels of the huntingtin protein (HTT) in people with the neurodegenerative disorder. That’s according to interim three-month data from the Phase 2a PIVOT-HD trial (NCT05358717), which found that use of the investigational oral therapy did not…

Scientists have figured out the first molecular events in the formation of toxic huntingtin protein clumps in Huntington’s disease. The findings shed light about why only mutations that result in at least 36 repeats of glutamine (one of protein’s building blocks) in the huntingtin protein cause the disease —…