Article

Muscle microRNAs in the cerebrospinal fluid predict clinical response to nusinersen therapy in type II and type III spinal muscular atrophy patients

2021-07-31

Abstract excerpt

<h4>Objective</h4> The antisense oligonucleotide nusinersen (spinraza) regulates splicing of the survival motor neuron 2 ( SMN2) messenger RNA to increase SMN protein expression and has improved ventilator free survival and motor function outcomes in infantile onset forms of SMA, treated early in the course of the disease. However, the response in later onset forms of SMA is highly variable and dependent on sympto...

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Literature Corpus work: bfe8d89d-1282-5d0f-87fc-f20ba356e80a
DOI: 10.1101/2021.07.29.21261322

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