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Nat Rev Drug Discov. 2013 Apr;12(4):287-305. doi: 10.1038/nrd3831. Epub 2013 Mar 15.

Animal models for metabolic, neuromuscular and ophthalmological rare diseases.

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Human Medicines Special Areas, Human Medicines Development and Evaluation, European Medicines Agency, London E14 4HB, UK.


Animal models are important tools in the discovery and development of treatments for rare diseases, particularly given the small populations of patients in which to evaluate therapeutic candidates. Here, we provide a compilation of mammalian animal models for metabolic, neuromuscular and ophthalmological orphan-designated conditions based on information gathered by the European Medicines Agency's Committee for Orphan Medicinal Products (COMP) since its establishment in 2000, as well as from a review of the literature. We discuss 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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