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.
Animal models for metabolic, neuromuscular and ophthalmological rare diseases
Published by Macmillan Publishers Limited
Animal ModelFeline ModelInfrafrontierMouse ModelNeuromuscular DiseaseOphtalmological Disease