U.K. Rare Diseases Framework, a government-led approach to raising national awareness of rare disorders, accelerating diagnoses, and improving patient care and treatment, is now in practice. This framework, signed by all four countries take make up the U.K. — England, Scotland, Wales and Northern Ireland — builds on the success…
Triplet Therapeutics announced it has completed patient enrollment in SHIELD HD, a natural history study documenting the course of Huntington’s in the absence of treatment and associated disease biomarkers. With more than 60 people taking part, SHIELD HD (NCT04406636) has attained and exceeded its target enrollment goal ahead…
No significant safety concerns associated with AMT-130, uniQure’s experimental gene therapy for Huntington’s disease, were identified in the first two patients participating in a Phase 1/2 trial assessing the treatment’s safety, tolerability, and efficacy. These were the main conclusions that came out of the first meeting of an…
An enzyme called TANK‐binding kinase 1 (TBK1) can lower levels of the mutant huntingtin protein — which underlies the development of Huntington’s disease — and prevent its aggregation in cellular and animal models of the disease, a study reports. Data showed that TBK1 is able to do this by…
The length of CAG repeats within the huntingtin (HTT) gene tends to increase as people with Huntington’s disease get older and is directly correlated with poor clinical outcomes, a study has found. The study, “A genetic association study of glutamine-encoding DNA sequence structures, somatic CAG expansion, and…
Mutations associated with Huntington’s disease increase nerve cells’ resistance to high levels of manganese, according to a recent study. The results of the study, “Huntington’s disease associated resistance to Mn neurotoxicity is neurodevelopmental stage and neuronal lineage dependent,” were published in NeuroToxicology. Manganese (Mn) is…
Different types of apathy — a common psychiatric symptom of Huntington’s disease — are associated with specific alterations in the brain’s white matter in patients at different stages of the disease, a study finds. White matter refers to regions of the brain made up of myelinated…
Laquinimod, an experimental immunomodulatory therapy developed by Active Biotech and Teva Pharmaceuticals, reduced signs of inflammation and brain atrophy (shrinkage) in patients with Huntington’s disease, data from an exploratory analysis of a Phase 2 trial show. The new data were presented by Active Biotech in three posters…
Huntington’s disease may manifest itself for the first time during neurulation, a process in which the neural tube — the precursor of our nervous system (brain and spinal cord) — develops while an embryo is forming, scientists report. These findings are in the study “Self-organizing neuruloids…
Patients at a pre-symptomatic stage of Huntington’s disease have marked difficulties recognizing and understanding negative and neutral mental states of other individuals, regardless of gender, a study has found. The results, “Effects of Stimulus-Related Variables on Mental States Recognition in Huntington’s Disease,” were published in the…
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