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While SMN restoration therapies are beneficial, they are not a cure. We aimed to identify potentially novel treatments to alleviate muscle pathology combining transcriptomics, proteomics, and perturbational data sets. This revealed potential drug candidates for repurposing in SMA. One of the candidates, harmine, was further investigated in cell and animal models, improving multiple disease phenotypes, including lifespan, weight, and key molecular networks in skeletal muscle. Our work highlights the potential of multiple and parallel data-driven approaches for the development of potentially novel treatments for use in combination with SMN restoration therapies.<\/jats:p>","DOI":"10.1172\/jci.insight.149446","type":"journal-article","created":{"date-parts":[[2021,7,7]],"date-time":"2021-07-07T14:01:34Z","timestamp":1625666494000},"source":"Crossref","is-referenced-by-count":22,"title":["Combining multiomics and drug perturbation profiles to identify muscle-specific treatments for spinal muscular atrophy"],"prefix":"10.1172","volume":"6","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0151-2983","authenticated-orcid":true,"given":"Katharina E.","family":"Meijboom","sequence":"first","affiliation":[{"name":"Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, United Kingdom."},{"name":"Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts, 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