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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Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT)

Published by The Company of Biologists Ltd

Mouse modelsNeuromuscular diseasesTREAT-NMD

This Review evaluates the experience accumulated by the TREAT-NMD Advisory Committee for Therapeutics, which provides detailed constructive feedback on clinical proposals for neuromuscular diseases. It emphasizes that a timely critical review of preclinical efficacy data from animal models, including biomarkers for specific diseases, combined with adherence to existing guidelines and standard protocols, can significantly help to de-risk clinical programs and prevent disappointments and costly engagement.

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Drosophila Genomics Resource Center

Published by n/a

Fruit Fly Model

The Drosophila Genomics Resource Center (DGRC) serves the Drosophila research community by: (a) collecting and distributing DNA clones and vectors; (b) collecting and distributing Drosophila cell lines; (c) developing and testing genomics technologies for use in Drosophila and assisting members of the research community in their use.

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