Key result
Recent advances in modelling neuromuscular disorders, particularly X-linked myotubular myopathy, aim to improve preclinical studies and potentiate clinical translation for novel therapeutics.
Highlights the importance of advanced preclinical models for neuromuscular disorders to improve clinical translation of novel therapeutics.
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.
No takes yet. Share an insight, caveat, or question.
Maaike van Putten (2022) conducted an editorial in Neuromuscular disorders (NMDs). Recent advances in modelling neuromuscular disorders, particularly X-linked myotubular myopathy, aim to improve preclinical studies and potentiate clinical translation for novel therapeutics.
Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context: