News

Alternative splicing, a molecular process that’s crucial for genes being “read” to produce proteins, is dysregulated in Huntington’s disease from the early stages of neuronal development, a study indicates. Notably, splicing changes were found to be dependent on the length of CAG repeats in the HTT gene — the cause…

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…

Folia Health is expanding the use of its patient-centered Folia app, designed to collect and manage patient-reported health data, to people with Huntington’s disease (HD). The free app lets patients with certain conditions — now including known or suspected Huntington’s — to record and visualize information about their…

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…

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,…