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• W1486242746 abstract "This chapter focuses on the utility of animal models in the development of drugs for rare diseases. Nonclinical safety evaluations are based on pharmacological activity, pharmacodynamics, pharmacokinetics and toxicology testing in relevant species. Animal models have significantly enhanced our understanding of several rare diseases and enabled the development of novel therapies. The appropriate animal disease model could be an invaluable tool to study the safety and efficacy of novel therapeutics for rare diseases such as Niemann-Pick disease, Lecithin-Cholesterol Acetly-transferase (LCAT) deficiency, Pompe disease, and Duchenne muscular dystrophy (DMD). Furthermore, the concept of “drug repurposing” has recently gained interest, whereby novel indications are explored for known mechanisms of action or are identified as new targets for known drugs." @default.
• W1486242746 created "2016-06-24" @default.
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• W1486242746 date "2013-07-31" @default.
• W1486242746 modified "2023-09-25" @default.
• W1486242746 title "Disease-Specific Models to Enhance Pediatric Drug Development" @default.
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• W1486242746 doi "https://doi.org/10.1002/9781118312087.ch22" @default.
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