Defects in cells’ “cleaning system,” known as autophagy, may underlie nerve cell degeneration in early stages of Huntington’s disease, according to a recent study. Treatments to improve this process in affected nerve cells could be a promising therapy for Huntington’s and other neurodegenerative diseases. The study,…
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Changes in the brain’s white matter in early Huntington’s disease can reflect a process that’s clinically relevant and might be linked to early cognitive dysfunction, according to researchers from Harvard Medical School. Their study, “Complex spatial and temporally defined myelin and axonal degeneration in Huntington disease,” was published in…
An investigational compound called VU0409551 could reverse memory deficiencies and improve learning, according to the results of a mouse study in Huntington’s disease. The study, “The mGluR5 positive allosteric modulator VU0409551 improves synaptic plasticity and memory of a mouse model of Huntington’s disease,” was published in the Journal…
Laquinimod Fails to Improve Motor Function but Reduces Brain Atrophy in Phase 2 Huntington’s Trial
Immunotherapy candidate laquinimod failed to meet its primary objective of improving motor function in Huntington’s disease patients after 12 months of treatment in a Phase 2 clinical trial. However, it did meet its secondary goal of reducing brain atrophy, according to a press release from Active Biotech. Laquinimod, developed…
PRIME Designation Should Help Promising Experimental Therapy Reach Huntington’s Patients Faster
The European Medicines Agency (EMA) has granted Priority Medicine (PRIME) designation to Roche’s investigational therapy RG6042 for the treatment of Huntington’s disease. PRIME provides a pathway for expedited assessment and review by the EMA, potentially enabling the drug to reach patients sooner. “We are very pleased…
Researchers have found a protein, called UBR5, that may reduce neurodegeneration in Huntington’s disease (HD). This protein prevents the toxic aggregation (clustering) of mutant huntingtin protein, which is the underlying mechanism that triggers nerve cells’ death in Huntington’s patients. The study, “The ubiquitin ligase UBR5 suppresses proteostasis collapse in pluripotent…
Damage to Nuclear DNA Shown to Correlate with Huntington’s Duration, Patient’s Ability to Function
The level of damage present in the DNA inside the cell nucleus — called nuclear DNA — correlates with disease duration and functional capacity in patients with Huntington’s disease, suggesting its potential use as a disease biomarker, a new study shows. The study, “Increased nuclear DNA damage precedes mitochondrial dysfunction in…
SOM3355 — a medicine approved for high blood pressure and chest pain — is being tested in a Phase 2 trial as a means to treat chorea in Huntington’s disease patients, SOM Biotech announced. The proof-of-concept study (NCT03575676) will recruit participants in four clinical sites in or near Barcelona, Spain.
Targeting specific genes involved in inflammation and cellular structure may be a therapeutic strategy to reduce protein clumps in patients with Huntington’s disease, according to researchers. Their study, “High-Throughput Functional Analysis Distinguishes Pathogenic, Nonpathogenic, and Compensatory Transcriptional Changes in Neurodegeneration,” appeared in the journal Cell…
Retrotope’s investigative therapy RT001 reduced cognition deficits in mice with Huntington’s disease, and was found to lower the amount of oxidative damage in their brains. The study, “Deuterium‐reinforced linoleic acid lowers lipid peroxidation and mitigates cognitive impairment in the Q140 knock in mouse model of Huntington’s disease,” was published in…
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