Dutch pharmaceutical company uniQure says it plans to conduct preclinical studies leading to clinical testing of a gene therapy in patients with Huntington’s disease. The company is developing AMT-130, an experimental therapy that aims to block production of the mutated and aggregation-prone huntingtin protein in patients. Composed of a small…
Researchers used gene editing to suppress the activity of protein mutations in the brains of mice with  Huntington’s disease. Suppressing the huntingtin protein alleviated early signs of the disease and reduced the mice’s movement problems, the study reported. The research, “CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of…
Therapies aimed at reducing the levels of a protein called HDAC3 may improve cognitive decline in Huntington’s disease, according to the results of a new study. The study titled, “A selective inhibitor of histone deacetylase 3 prevents cognitive deficits and suppresses…
A snapshot of a key brain receptor in action might advance the understanding of disease processes in Huntington’s disease and allow scientists to identify new treatments, researchers at Columbia University Medical Center (CUMC) hope. Their work, published in the journal Nature, describes how the neurotransmitter glutamate binds to…
Researchers have created the first freely available data network — called HDNetDB — that aims to advance research into Huntington’s disease and speed up drug discovery for the incurable condition. The approach, described in an article in the journal Scientific Reports, particularly focuses on the complex molecular workings…
Wave Life Sciences will start two Phase 1b/2a clinical trials to investigate the safety and tolerability of its products WVE-120101 and WVE-120102 in Huntington’s disease patients. The trials will also look at the therapies’ effectiveness. “Wave’s PRECISION-HD program is the first to target the underlying cause of Huntington’s disease…
Huntington’s patients who are taking Xenazine for the twitching condition known as chorea can switch without concern to the newly approved Austedo, according to Austedo’s maker, Teva Pharmaceutical Industries. A Phase 3 clinical trial showed that the switch was safe and that Austedo (deutetrabenazine) did a better job of controlling chorea than…
A sluggish metabolism in the cells of the striatum, a brain region essential for motor function control, is associated with disease severity in Huntington’s patients, while a higher metabolism in other brain regions seems to be associated with more severe motor symptoms in patients, according to researchers. These findings…
Mutations of the MSH3 gene may be associated with the progression of Huntington’s disease, according to a British study. The gene could become a biomarker for the disease, and scientists may be able to develop Huntington’s therapies around it, the researchers from University College London and Wales’ Cardiff University said.
Mitoconix Bio has secured $20 million in financing to continue developing MTC-1203Â as a treatment for mitochondrial impairment in Huntington’s and Parkinson’s diseases. Several pharmaceutical companies and medical-related venture-capital funds participated in the financing. They include Aril Bioscience, OrbiMed, Remiges Ventures and the Dementia Discovery Fund. “We are very…
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