The predictive value of models of neuromuscular disorders to potentiate clinical translation
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...
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The Company of Biologists,
2022-07-01T00:00:00Z.
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| 042 | |a dc | ||
| 100 | 1 | 0 | |a Maaike van Putten |e author |
| 245 | 0 | 0 | |a The predictive value of models of neuromuscular disorders to potentiate clinical translation |
| 260 | |b The Company of Biologists, |c 2022-07-01T00:00:00Z. | ||
| 500 | |a 1754-8403 | ||
| 500 | |a 1754-8411 | ||
| 500 | |a 10.1242/dmm.049788 | ||
| 520 | |a 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. | ||
| 546 | |a EN | ||
| 690 | |a Medicine | ||
| 690 | |a R | ||
| 690 | |a Pathology | ||
| 690 | |a RB1-214 | ||
| 655 | 7 | |a article |2 local | |
| 786 | 0 | |n Disease Models & Mechanisms, Vol 15, Iss 7 (2022) | |
| 787 | 0 | |n http://dmm.biologists.org/content/15/7/dmm049788 | |
| 787 | 0 | |n https://doaj.org/toc/1754-8403 | |
| 787 | 0 | |n https://doaj.org/toc/1754-8411 | |
| 856 | 4 | 1 | |u https://doaj.org/article/8ec2d06e01fc457ba19f76a31a33b615 |z Connect to this object online. |