Article

RNA splicing modulator induces peripheral neuropathy with increased neurofilament light chain levels via p53 signaling

2023-08-05

Abstract excerpt

RNA splicing modulators are a new class of small molecules with the potential to modify the expression levels of proteins. A recent clinical trial investigating the splicing modulator branaplam for Huntington’s disease to lower huntingtin levels was terminated due to the development of peripheral neuropathy. Here, we describe how branaplam leads to this adverse effect. On a cellular level, branaplam disrupts neuri...

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Literature Corpus work: f09d1d25-91c0-51a3-aeef-d3b77dcf0273
DOI: 10.1101/2023.08.02.551640

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