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- W7655099 abstract "Enormous advances in life sciences and molecular biology mean that research today generates massive amounts of data that can potentially serve to advance the development of individualized therapies. Such treatments are based on the principles of disease prevention, prediction, early diagnosis and reliable monitoring of therapeutic efficacy with the goal of providing personalized treatments that improve the health of individuals. The development of personalized medicine is of considerable importance for pediatric patient populations, and represents a move away from the use of treatment dosages based on experience with the same compounds in adults. Currently, however, we know little about developmental pharmacogenomics and, although many biomarkers are available for use in clinical research, there have been few applications in the management of pediatric diseases. This chapter reviews the requirements for pediatric personalized medicine and describes a group of diseases that require such an approach. Personalized medicine is particularly relevant for the treatment of rare childhood diseases, and the group of life-threatening neurological diseases known as lysosomal storage diseases (LSDs) represents a potential study population. The genetic bases of these disorders are generally well defined, there is the potential for diagnosis at birth or prenatally, and there are several therapeutic options available or under development." @default.
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- W7655099 date "2012-01-05" @default.
- W7655099 modified "2023-09-26" @default.
- W7655099 title "Personalized Medicine in Rare Pediatric Neurometabolic Diseases" @default.
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- W7655099 doi "https://doi.org/10.1007/978-94-007-5866-7_14" @default.
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