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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KNOCKOUTS MODEL THE 100 BEST-SELLING DRUGS — WILL THEY MODEL THE NEXT 100?

Published by Nature

Knock-Out Mouse Model

this article is a retrospective evaluation of the knockout phenotypes for the targets of the 100 best-selling drugs indicates that these phenotypes correlate well with known drug efficacy, illuminating a productive path forward for discovering future drug targets

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Nonhuman Primate Models in the Genomic Era: A Paradigm Shift

Published by ILAR J.

Non-Human Primate Model

This article describes the genetic and genomic revolution thath is changing the paradigm of the nonhuman primates as a disease model and the new mandates on how nonhuman primates are used in biomedical research.

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CCAC guidelines: Nonhuman primates

Published by CCAC

NHPNonhuman primates

The CCAC guidelines: Nonhuman primates provides information for investigators, animal care committees, facility managers, veterinarians and animal care staff to help facilitate improvement in both the care given to nonhuman primates and the manner in which experimental procedures are carried out

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Enhancing translation: guidelines for standard pre-clinical experiments in mdx mice

Published by Neuromuscular Disorders

MiceNeuromuscular diseasesTREAT-NMD

The authors propose the introduction of a standard study design for the mdx mouse model. Several aspects, including animal care, sampling times and choice of tissues, as well as recommended endpoints and methodologies are addressed and, for each aspect, a standard procedure is proposed. Testing of all new molecules/drugs using a widely accepted and agreed upon standard experimental protocol would greatly improve the power of pre-clinical experimentations and help identifying promising therapies for the translation into clinical trials for boys with Duchenne Muscular Dystrophy

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