Ionis Pharmaceuticals has completed enrollment of the Phase 1/2a study evaluating the safety, tolerability, properties and movement within the body of Ionis-HTTRx in patients with Huntington’s disease (HD). Patients who complete the randomized, placebo-controlled, dose escalation study (NCT02519036) will be eligible to enroll in an open-label…
Researchers have identified a variant in the gene MSH3 that is linked with Huntington’s disease (HD) progression. The study, “Identification of genetic variants associated with Huntington’s disease progression: a genome-wide association study,” was published in the journal The Lancet Neurology. TRACK-HD was a prospective and observational study which…
Inhibiting the activity of an enzyme preserved the movement capabilities of mice with Huntington’s, slowed the progression of the disease and extended the animals’ lives, according to a study. The dSETDB1/ESET enzyme can disrupt a mechanism that controls normal gene expression, the researchers discovered. Alterations in that expression, along with…
Low levels of a protein called Pyk2 may be associated with the development of Huntington’s disease, according to a new study. Researchers found that normalizing its levels in the hippocampus, the brain region responsible for learning and memory, rescues memory deficits. These findings were published in a study titled, “…
A factor called neurofilament light (NfL) may act as a biomarker of progression in Huntington’s disease, as blood levels of the molecule increase with more severe neurodegeneration. However, the factor also might offer a better means to predict disease onset in people carrying huntingtin mutations, as demonstrated in the study,…
A mutated form of the huntingtin (mHTT) protein has been acknowledged as the cause of Huntington’s disease, but researchers have not been fully clear about how it disrupts the function of brain cells. A study, “Mutant Huntingtin Disrupts the Nuclear Pore Complex,” that was published in the scientific journal…
Voyager Therapeutics has a new candidate for the treatment of Huntington’s disease. The selected gene therapy is called VY-HTT01 and aims to prevent the production of protein from the mutant huntingtin gene (HTT). The compound is now undergoing preclinical studies of drug characteristics and toxicology as the company plans to file…
Researchers at the University of California and the Salk Institute for Biological Studies have identified the mechanisms that make the brain’s immune cells different from all other immune cells A finding with implications for Huntington’s disease was that genes associated with neurological illnesses have higher lessons of expression in microglia,…
Abnormal proteins found in neurodegenerative diseases such as Alzheimer’s, Parkinson’s, and Huntington’s seem to rely on the same mechanism to spread within the brain and disrupt its normal functions, according to a study published in the journal Acta Neuropathologica. The cellular process described in the study, “Endocytic vesicle rupture…
A team of researchers has discovered new insights into cell processes that have gone awry in Huntingon’s disease (HD) patients. This discovery could point scientists toward a potential method of interfering with those processes. The study, “KEAP1-modifying small molecule reveals muted NRF2 signaling responses in neural stem cells from Huntington’s…
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