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This highlights the need for targeted therapies that address these evolving cardiac pathologies. Musculopathies by and large lack cures that directly target the genetic basis of the diseases; however, as our understanding of the genetic causes of these conditions has evolved, it has become tractable to develop targeted therapies using biologics, to design precision approaches to target the primary genetic causes of these varied diseases. Using the examples of Duchenne muscular dystrophy, Friedreich ataxia and Pompe disease, we discuss how the genetic causes of such diseases derail diverse homeostatic, energetic and signalling pathways, which span multiple cellular systems in varied tissues across the body. We outline existing therapeutics and treatments in the context of emerging novel genetic approaches. We discuss the hurdles that the field must overcome to deliver targeted therapies across the many tissue types affected in primary myopathies.<\/jats:p>","DOI":"10.1113\/ep090436","type":"journal-article","created":{"date-parts":[[2023,12,14]],"date-time":"2023-12-14T16:07:21Z","timestamp":1702570041000},"page":"175-189","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Targeted genetic therapies for inherited disorders that affect both cardiac and skeletal muscle"],"prefix":"10.1113","volume":"109","author":[{"given":"Yiangos","family":"Psaras","sequence":"first","affiliation":[{"name":"Division of Cardiovascular Medicine Radcliffe Department of Medicine University of Oxford  Oxford UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4671-2030","authenticated-orcid":false,"given":"Christopher N.","family":"Toepfer","sequence":"additional","affiliation":[{"name":"Division of Cardiovascular Medicine Radcliffe Department of Medicine University of Oxford  Oxford UK"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2023,12,14]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1002\/humu.20918"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.27604"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1101\/gr.123463.111"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41586-019-1711-4"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2019.06.024"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0168-9525(03)00081-7"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.276.5319.1709"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2020.08.018"},{"key":"e_1_2_9_10_1","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1186\/s12968-016-0311-9","article-title":"Pattern and prognostic value of cardiac involvement in patients with late\u2010onset pompe disease: A comprehensive cardiovascular magnetic resonance approach","volume":"18","author":"Boentert M.","year":"2016","journal-title":"Journal of Cardiovascular Magnetic Resonance"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1042\/BCJ20200331"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1021\/acs.analchem.5b00169"},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-017-0693-2"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/6.11.1771"},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.271.5254.1423"},{"key":"e_1_2_9_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/jimd.12560"},{"key":"e_1_2_9_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(11)60756-3"},{"key":"e_1_2_9_18_1","doi-asserted-by":"crossref","unstructured":"Corben L. 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