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Management requires a multidisciplinary approach to the organ\u2010specific manifestations including specialists from cardiology, hematology, nutrition, physical therapy, genetics, and metabolism. Currently, treatment is centered on management of specific clinical features, and is not targeted toward remediating the underlying biochemical defect. However, two clinical trials have been recently undertaken which target the mitochondrial pathology of this disease: a study to examine the effects of elamipretide, a cardiolipin targeted agent, and a study to examine the effects of bezafibrate, a peroxisome proliferator\u2010activated receptor (PPAR) agonist. Treatments to directly target the defective TAFAZZIN pathway are under development, including enzyme and gene therapies.<\/jats:p>","DOI":"10.1002\/jimd.12453","type":"journal-article","created":{"date-parts":[[2021,10,29]],"date-time":"2021-10-29T08:15:15Z","timestamp":1635495315000},"page":"17-28","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":41,"title":["Current and future treatment approaches for Barth syndrome"],"prefix":"10.1002","volume":"45","author":[{"given":"Reid","family":"Thompson","sequence":"first","affiliation":[{"name":"Department of Pediatric Cardiology Johns Hopkins University School of Medicine  Baltimore Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John","family":"Jefferies","sequence":"additional","affiliation":[{"name":"The Cardiovascular Institute The University of Tennessee Health Science Center  Memphis Tennessee 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