Marisa Wexler, MS,  senior science writer—

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,…

The Phase 3 PROOF-HD trial failed to meet its main goal of showing that pridopidine, Prilenia Therapeutics’ experimental therapy, could slow functional decline compared with a placebo for adults with Huntington’s disease. However, preliminary top-line data indicated a potential slowing of disease progression among patients who were…

Asklepios Biopharmaceutical (AskBio) has launched a first-in-human clinical trial testing AB-1001 — an experimental gene therapy designed to modulate cholesterol metabolism in the brain — in people with Huntington’s disease. The trial is currently recruiting participants at a site in Paris. It is open to adults, ages 18…