Clumps of the mutant huntingtin protein, which are the known cause of Huntington’s disease, poke holes in the membrane that surrounds and protects cells’ DNA-filled nucleus, according to preclinical research. These protein aggregates had tiny, protruding fibers that damaged the membrane, which is called the nuclear envelope. Lab-grown cells…
News
Being physically active is significantly associated with a delayed age of the onset of Huntington’s disease and spending more time watching television is significantly linked to an earlier age of onset. These are the findings of a study that analyzed genetic variants linked with different levels of physical activity…
A high level of education, low to moderate alcohol intake, not smoking, and controlling one’s weight may slow Huntington’s disease progression among those who’ve yet to have symptoms, a study shows. “Reducing modifiable risk factors for [Huntington’s] is one way to support the presymptomatic population,” which is made up…
Levels of glial fibrillary acidic protein (GFAP), a marker of damage to specific neuron-supporting cells, in the fluid surrounding the brain and spinal cord increase with the progression of Huntington’s disease, a study showed. Higher GFAP levels in the cerebrospinal fluid (CSF) were also significantly associated with more severe…
Ingrezza Sprinkle, a new formulation of Ingrezza (valbenazine) that’s designed to be easier to swallow, is now available in the U.S, Neurocrine Biosciences, which markets the therapy, announced. Ingrezza Sprinkle comes in capsules that are meant to be opened and the contents sprinkled over soft foods like yogurt,…
The investigational gene therapy AMT-130 is slowing disease progression for people with early-stage Huntington’s disease, as well as lowering levels of neurofilament light chain (NfL), a marker of nerve cell damage. That’s according to two-year data from two Phase 1/2 clinical trials: one in the U.S. (NCT04120493)…
More than three months of treatment with WVE-003 safely leads to significant reductions in mutant huntingtin — the protein that toxically accumulates in Huntington’s disease — for people with early Huntington’s, while preserving levels of the healthy version of huntingtin and trends of slower brain tissue loss and clinical…
Neurocrine Biosciences is offering a new online educational resource called More Than HD that seeks to inspire and give hope to Huntington’s disease patients and their caregivers by helping them prepare for their journey with the disease. “It was important for us at Neurocrine to create More Than…
An investigational oral therapy from PTC Therapeutics called PTC518 safely and effectively reduces levels of the mutant huntingtin protein (mHTT) in Huntington’s disease patients, while being linked to trends of slower disease progression. That’s according to one-year interim data from the ongoing Phase 2a PIVOT-HD clinical trial…
Significant impairments in cognitive function are evident in people with Huntington’s disease relative to healthy adults, as assessed with a disease-specific battery of cognitive tests, top-line data from a Phase 2 clinical trial show, meeting its main goal. The two-part trial, called SURVEYOR (NCT05358821), also evaluated the safety…
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