Article

A transcriptomics-based drug repositioning approach to identify drugs with similar activities for the treatment of muscle pathologies in spinal muscular atrophy (SMA) models

2023-06-14

Abstract excerpt

<h4>ABSTRACT</h4> Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder caused by the reduction of survival of motor neuron (SMN) protein levels. Although three SMN-augmentation therapies are clinically approved that significantly slow down disease progression, they are unfortunately not cures. Thus, complementary SMN-independent therapies that can target key SMA pathologies and that can support the c...

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Literature Corpus work: dfdd715e-ed15-53e4-a8df-570f295c9b34
DOI: 10.1101/2023.06.14.544899

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