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Huntington's disease (HD) is a neurodegenerative disease caused by the autosomal dominant amplification of the cytidine-adenine (CAG) fragment in the Huntington (HTT) gene, resulting in the gradual decline of motor, cognitive and mental functions As a feature
HD was first marked by a long asymptomatic pre-stage, which lasted for decades, followed by a comprehensive manifestation of the severity of the disease
Therefore, one of the most critical obstacles to effective new clinical trials is how to assign heterogeneous individuals to homogeneous subgroups
Finding the best window for future drug intervention and determining effective HD stratification will be of great significance to the development of treatment
This method can conduct research on a more representative sample of the HD population and conduct cross-study verification to improve the robustness of the results
In this way, Pubu M.
They combined the clinical and morphological imaging data of 1082 participants in the IMAGE-HD, TRACK-HD, and PREDICT-HD studies, with a longitudinal duration of 1-10 years
They found that the volume of the putamen and the caudate nucleus group can distinguish the age group of CAG, and it is the best variable that can reflect the progress
If only the volume of the caudate nucleus is used, the statistical model can best describe the advanced disease progression in the comprehensive data
If only the volume of the caudate nucleus is used, the statistical model can best describe the advanced disease progression in the comprehensive data
The important significance of this study lies in the discovery: During the entire study process, monitoring volume changes, together with primary and secondary clinical endpoints, helps increase people’s awareness of the improvement of functional outcomes, and helps to improve clinical trials.
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