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…
Based on MRI scans, Huntington’s disease patients with major cognitive impairments have reduced gray matter volume and a thinner brain cortex than those without cognitive problems, a study has demonstrated. Major cognitive decline was not associated with CAG repeat length, age, or education, suggesting additional…
A single injection of the protein CDNF — cerebral dopamine neurotrophic factor — into the brains of rats with induced Huntington’s disease protected affected nerve cells from degeneration and improved motor performance, a study reported. These findings support further development of CDNF as a treatment…
Administration of the hormone erythropoietin (EPO) using a gene transfer method improved spatial cognition, stimulated the branching of nerve stem cells, and preserved the brain region known as the hippocampus in a mouse model of…
Teva Pharmaceuticals’ Austedo (deutetrabenazine tablets) has been approved by China’s National Medical Products Administration (NMPA) for treating chorea associated with Huntington’s disease and tardive dyskinesia…
A DNA-binding compound can reverse the repeat expansion mutation that is associated with Huntington’s disease, an early study reported. This newly developed small molecule was also seen to reduce disease-causing protein aggregates in nerve cells within the brains of mice in a disease model,…
In a proof-of-principle experiment, a new gene therapy approach with the potential to treat people with Huntington’s disease has been shown to effectively convert astrocyte brain cells of mice into functioning neurons. Those neurons took the place of nerve cells in the mice that were damaged by…
A potential treatment called RG6042, which target all forms of the mutant huntingtin protein (mHTT) — the underlying cause of Huntington’s disease, has been named an orphan drug by the Ministry of Health,…
Neural stem cell researchers at the University of California, Irvine, (UCI) will collaborate with AgeX Therapeutics to develop cell-based therapies to treat Huntington’s disease and other neurological disorders. Leslie Thompson…
Raman spectroscopy, an analysis technique that uses light, identified a specific molecular fingerprint in the blood of people with Huntington’s disease, a study has found. This technique may be useful as a non-invasive diagnostic tool to track disease progression and as…
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