Deficits in motor function are a hallmark of Huntington disease (HD). The Unified Huntington's Disease Rating Scale Total Motor Score (UHDRS-TMS) is a categoric clinical rating scale assessing multiple domains of motor disability in HD. The UHDRS-TMS or subsets of its items have served as primary or secondary endpoints in numerous clinical trials. In spite of a well-established video-based annual online certification system, intra- and interrater variability, subjective error, and rater-induced placebo effects remain a concern. In addition, the UHDRS-TMS was designed to primarily assess motor symptoms in manifest HD. Recently, advancement of technology resulted in the introduction of the objective Q-Motor (i.e., Quantitative-Motor) assessments in biomarker studies and clinical trials in HD. Q-Motor measures detected motor signs in blinded cross-sectional and longitudinal analyses of manifest, prodromal, and premanifest HD cohorts up to two decades before clinical diagnosis. In a multicenter clinical trial in HD, Q-Motor measures were more sensitive than the UHDRS-TMS and exhibited no placebo effects. Thus, Q-Motor measures are currently explored in several multicenter trials targeting both symptomatic and disease-modifying mechanisms. They may supplement the UHDRS-TMS, increase the sensitivity and reliability in proof-of-concept studies, and open the door for phenotype assessments in clinical trials in prodromal and premanifest HD.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1016/B978-0-12-801893-4.00018-3 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Effect of early administration of fibrinogen replacement therapy in traumatic haemorrhage: a systematic review and meta-analysis of randomised controlled trials with narrative synthesis of observational studies.
Crit Care
January 2025
Centre for Trauma Sciences, Blizard Institute, Queen Mary University of London, London, England.
Background: In severely injured trauma patients, hypofibrinoginaemia is associated with increased mortality. There is no evidence-based consensus for what constitutes optimal fibrinogen therapy, treatment dose or timing of administration. The aim of this systematic review was to evaluate the effects of early fibrinogen replacement, either cryoprecipitate or fibrinogen concentrate (FgC) on mortality, transfusion requirements and deep venous thrombosis (DVT).
View Article and Find Full Text PDFExperiences, perceptions and ethical considerations of the malaria infection study in Thailand.
BMC Med Ethics
January 2025
Mahidol Oxford Tropical Medicine Research Unit, Faculty of Tropical Medicine, Mahidol University, 420/6 Rajvithi Road, Thunphayathai, Bangkok, 10400, Thailand.
Background: Thailand has made significant progress in malaria control efforts in the past decade, with a decline in the number of reported cases. However, due to cross-border movements over the past 5 years, reported malaria cases in Thailand have risen. The Malaria Infection Study in Thailand (MIST) involves deliberate infection of healthy volunteers with Plasmodium vivax malaria parasites, and the assessment of the efficacy of potential vaccine and drug candidates in order to understand acquired protection against malaria parasites.
View Article and Find Full Text PDFRationale and design of the HOVON 170 DLBCL-ANTICIPATE trial: preventing anthracycline-induced cardiac dysfunction with dexrazoxane.
Cardiooncology
January 2025
Department of Hematology, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.
Background: Dexrazoxane has been studied for its ability to prevent anthracycline-induced cardiac dysfunction (AICD) in several trials but its use in clinical practice remains limited. This is related to the low to moderate quality of the generated evidence, safety concerns and restricted prescribing indications. Additional randomized trials are needed before this drug can be routinely integrated into cardio-oncology clinical practice.
View Article and Find Full Text PDFC-28 linker length modulates the activity of second-generation HIV-1 maturation inhibitors.
Virol J
January 2025
Virology Laboratory, Faculty of Life Sciences and Biotechnology, South Asian University (SAU), New Delhi, 110068, India.
Maturation inhibitors (MIs) block HIV-1 maturation by preventing the cleavage of the capsid protein and spacer peptide 1 (CA-SP1). Bevirimat (BVM), a first-in-class MI, displayed sub-optimal efficacy in clinical trials due to presence of SP1:V7A polymorphism in the Gag protein.This polymorphism is inherently present in HIV-1 subtype C and conferred resistance to BVM.
View Article and Find Full Text PDFLevilactobacillus brevis CD2 as a multifaceted probiotic to preserve oral health: results of a double-blind, randomized, placebo-controlled trial in healthy adults.
J Transl Med
January 2025
Department of Life, Health & Environmental Sciences, University of L'Aquila, Building Rita Levi Montalcini, Coppito, L'Aquila, 67100, Italy.
Background: A growing number of in vitro and in vivo studies suggest the application of probiotics as a natural approach to maintaining oral health. This double-blind, randomized controlled trial aimed to evaluate the efficacy of Levilactobacillus brevis CD2 (CNCM I-5566), a multifunctional probiotic frequently used in oral medicine, in preserving or improving several recognized oral health indicators.
Methods: Thirty consenting healthy adults were randomized to receive four lozenges per day of L.
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!