Marisa Wexler, MS,  senior science writer—

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to SAGE-718, Sage Therapeutics’ experimental oral therapy for Huntington’s disease. The FDA gives this designation to therapies that have the potential to treat rare diseases, which are defined as conditions that affect fewer than 200,000 people…

The recent approval in the U.S. of Ingrezza (valbenazine) for adults with Huntington’s disease-associated chorea means patients now have access to a treatment option that’s likely easier to take, according to the chief medical officer of Neurocrine Biosciences, the therapy’s maker. “Ingrezza is always one capsule,…

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…

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 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…

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…

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 —…

The oral therapy valbenazine significantly reduces involuntary muscle contractions, or chorea, and patient-reported disease burden in people with Huntington’s disease, according to results from the Phase 3 KINECT-HD clinical trial. Top-line data were announced by Neurocrine Biosciences, the therapy’s manufacturer, in 2021. The full findings have…

Austedo XR, an extended-release oral formulation of Austedo (deutetrabenazine), is now commercially available in the U.S. as a treatment for adults with chorea, or uncontrolled muscle contractions, associated with Huntington’s disease. The new formulation is available in tablet strengths of 6 mg, 12 mg, and 24 mg,…