The predictive value of models of neuromuscular disorders to potentiate clinical translation

Putten, M. van (2022)

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Neuromuscular disorders (NMDs) are a heterogenous group of rare inherited diseases that compromise the function of peripheral nerves and/or muscles. With limited treatment options available, there is a growing need to design effective preclinical studies that can lead to greater success in clinical trials for novel therapeutics. Here, I discuss recent advances in modelling NMDs to improve preclinical studies as well as two articles from this issue that work in parallel to enable a deeper understanding of a particularly rare NMD, known as X-linked myotubular myopathy.

• All authors: Putten, M. van
• Date: 2022-07-01
• Title of host publication: Disease Models and Mechanisms
• Volume: 15
• Issue: 7
• DOI: doi:10.1242/dmm.049788