Biomarkers for Huntington's Disease
Improving Clinical Outcomes
Produktform: Buch / Einband - fest (Hardcover)
Huntington’s disease (HD) is a fatal, inherited, neurodegenerative disorder, characterized by chorea, motor instabilities, psychiatric manifestations and cognitive decline. George Huntington, an American physician, was the first person to provide a comprehensive description of adult-onset HD in 1872, including its autosomal-dominant pattern of inheritance. The HD gene, Huntingtin (HTT), was the first disease-associated gene to be mapped to a human chromosome, in 1983. Ten years later, researchers identified the disease mutation: an expanded triplet nucleotide repeat within exon 1 of the HTT gene. This mutation can now easily be measured by genetic testing, which can be used to confirm the genetic status of individuals at risk of developing HD. Early genetic testing provides an opportunity for clinical interventions aimed at delaying onset and/or slowing progression of disease; however, current treatments for HD are limited, with only two FDA-approved drugs available to manage chorea. Encouragingly, however, several disease-modifying treatment approaches are in the therapeutic pipeline, with 200 clinical studies, and many more preclinical studies, in the works. Robust and reliable biomarkers are needed to predict disease onset, monitor disease progression and assess treatment responses. More specifically, biomarkers to stratify patients for clinical trials and biomarkers to track drug efficacy will certainly lead to improved clinical trial design and success. This book will represent the first book focused solely on biomarkers for HD and will represent a distinct resource that will be informative, not only for clinicians and those involved in clinical trial design, but also for a wide range of neurodegenerative disease researchers. This edited volume is written by top leaders in the field, and takes a cross-disciplinary approach to cover a broad spectrum of biomarker types, in order to provide the latest advances in the development of biochemical, molecular, imaging and digital biomarkers that have been investigated for HD. With the ultimate goal of treating patients, the development of disease-associated biomarkers has never been more important.weiterlesen
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