A Genetic Lottery - a Column by Becky Field

The U.S. Food and Drug Administration (FDA) will review Neurocrine Biosciences’ application of a new oral granule formulation of Ingrezza (valbenazine) for involuntary muscle contractions, or chorea, in adults with Huntington’s disease. The therapy was recently approved in the U.S. for Huntington’s-associated chorea in the form…

The benefits of Ingrezza (valbenazine) for people with Huntington’s disease — reductions in uncontrolled movements and gains in clinician- and patient-reported health — were seen two weeks after starting treatment and maintained for up to three months, according to new KINECT-HD trial analyses. Ingrezza was cleared last month…

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…

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…

A computer-based test called SelfCog, which tests various cognitive functions in a standardized manner, yielded promising results in assessing cognitive decline in people with early-stage Huntington’s disease, a study has found. SelfCog demonstrated sensitivity to detect cognitive decline over a one-year follow-up, outperforming traditional cognitive assessments, and showed associations…