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Animal models for metabolic, neuromuscular and ophthalmological rare diseases

Published by Macmillan Publishers Limited

Animal ModelFeline ModelInfrafrontierMouse ModelNeuromuscular DiseaseOphtalmological Disease

the article provides a compilation of mammalian animal
models for metabolic, neuromuscular and ophthalmological orphan-designated conditions
based on information gathered by the EMA’s Committee for Orphan Medicinal Products (COMP). It is discussed the predictive value of the models and their advantages and limitations with the aim of highlighting those that are appropriate for the preclinical evaluation of novel
therapies, thereby facilitating further drug development for rare diseases.

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analysis of the attrition of drug candidates from four major pharmaceutical companies

Published by Macmillan Publishers Limited

Drug DiscoverySafety IssueSmall Molecule

This paper describes the compilation and analysis of combined data on the attrition of drug candidates from AstraZeneca, Eli Lilly and Company, GlaxoSmithKline and Pfizer. The analysis reaffirms that control of physicochemical properties during compound optimization is beneficial in identifying compounds of candidate drug quality and indicates for the first time a link between the physicochemical properties of compounds and clinical failure due to Safety Issues.

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