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

ABSTRACT 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...

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Bibliographic Details
Published inDisease models & mechanisms Vol. 15; no. 7
Main Author van Putten, Maaike
Format Journal Article
LanguageEnglish
Published Cambridge The Company of Biologists Ltd 01.07.2022
The Company of Biologists
Subjects
Clinical trials
Congenital diseases
Genes
Hereditary diseases
Insects
Muscles
Muscular dystrophy
Mutation
Myopathy
Natural history
Neuromuscular diseases
Pathology
Pathophysiology
Peripheral nerves
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Summary:ABSTRACT 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.
Bibliography:SourceType-Other Sources-1
content type line 63
ObjectType-Editorial-2
ObjectType-Commentary-1
ISSN:1754-8403
1754-8411
DOI:10.1242/dmm.049788