Application of precision medicine in rare neurodegenerative diseases / 国际药学研究杂志

ABSTRACT

Technological development has paved the way for accelerated genomic discovery and brought precision medicine into view. Genetics, bioinformatics, molecular imaging and management provide tools for realizing the idea of precision medicine. The goal of precision medicine is to deliver optimally targeted and timed interventions tailored to an individual’s molecular drivers of disease. It is very important for the implementation of precision medicine in rare neurodegenerative diseases, which are difficult to diagnose and treat. Precision medicine contributes to the identification of causative genes, the classification of genotypes and phenotypes of diseases, discovery of biomarkers with high sensitivity and specificity, and development of promising modified therapies. However, like the disadvantage encountered in other diseases, implementation of precision medicine in rare neurodegenerative diseases still confronts numerous challenges.