Steve Bryson, PhD, science writer —

The European Medicines Agency (EMA) has granted orphan drug status to SAGE-718 as a potential therapy for Huntington’s disease. The experimental therapy is being developed by Sage Therapeutics for treating cognitive impairment associated with Huntington’s and other neurological disorders. Orphan drug status is given to treatments with the potential to improve…

Neuron-targeted treatment based on zinc-finger DNA-binding proteins — called ZFPs — was found to lower brain levels of mutant huntingtin (mHTT) protein in a Huntington’s disease mouse model. The zinc-finger proteins rescued behavioral deficits and extended survival in these mice, the study demonstrated. ZFPs are among the most abundant…

The activity of focal adhesion kinase (FAK), an enzyme known to play an essential role in nerve cell function, is low in the brains of people with Huntington’s disease, researchers discovered. Cell and mouse models revealed that the presence of abnormal huntingtin protein, the underlying cause of Huntington’s, blocked…

Beyond genetic factors, the onset of Huntington’s disease later in life was related to being in a stable partnership, living in large cities, and having lower education levels, a large-scale analysis suggests. In comparison, a younger age at disease onset was associated with suicidal behavior and the use of…

A 56-year-old man with undiagnosed late-onset Huntington’s disease presented with Parkinson’s disease-like symptoms, including bradykinesia, or slowness of movement, a case study reported. He was correctly diagnosed after clinicians learned of a family history of Huntington’s and ordered genetic testing. Given the diagnosis, the researchers recommended that Huntington’s disease…

Activating the JAK2-STAT3 pathway in astrocyte cells reduced multiple hallmark features of Huntington’s disease  — including the toxic clumping of the huntingtin protein in nerve cells — from the brains of a mouse model, a study reported. “Our results open new therapeutic avenues to further enhance the natural partnership…

Clumps of protein resulting from HTT mutations, the underlying cause of Huntington’s disease, form distinct structures in different parts of the cell, according to detailed imaging analysis. The findings suggest these clumps form by different mechanisms and may require different therapeutic strategies to block their formation and toxicity that…

The perception of time is impaired in people with symptomatic Huntington’s disease in its early stages — but not in patients without symptoms, a study revealed. These findings suggest that time perception impairment may be used as a clinical tool for assessing those with the neurodegenerative disorder, the scientists…

Frequent bladder, bowel, and sexual problems were reported by people with Huntington’s disease, a study reveals. Recommended Reading October 13, 2021 Columns by B.J. Viau These 5 Changes Are…

No significant safety concerns were found in the first four patients participating in a Phase 1/2 trial assessing the safety and efficacy of uniQure’s AMT-130, an experimental gene therapy for Huntington’s disease. This…