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Elamipretide targets the inner mitochondrial membrane where it binds to cardiolipin, resulting in improved membrane stability, cellular respiration, and ATP production. In clinical trials, elamipretide produced clinical and functional improvements in adults and adolescents with mitochondrial disorders, such as primary mitochondrial myopathy and Barth syndrome; however, experience in younger patients is limited and to our knowledge, these are the first case reports on the safety and efficacy of elamipretide treatment in children under 12\u2009years of age. We describe the use of elamipretide in patients with mitochondrial disorders to provide dosing parameters in patients aged &lt;12\u2009years.<\/jats:p><\/jats:sec>","DOI":"10.1002\/jmd2.12335","type":"journal-article","created":{"date-parts":[[2022,9,21]],"date-time":"2022-09-21T23:10:43Z","timestamp":1663801843000},"page":"65-70","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Use of Elamipretide in patients assigned treatment in the compassionate use program: Case series in pediatric patients with rare orphan diseases"],"prefix":"10.1002","volume":"64","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-5126-8515","authenticated-orcid":false,"given":"Mary Kay","family":"Koenig","sequence":"first","affiliation":[{"name":"The University of Texas McGovern Medical School, Center for the Treatment of Pediatric Neurodegenerative Disease  Houston Texas USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sam Nick","family":"Russo","sequence":"additional","affiliation":[{"name":"The University of Texas McGovern Medical School, Center for the Treatment of Pediatric Neurodegenerative Disease  Houston Texas USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kim L.","family":"McBride","sequence":"additional","affiliation":[{"name":"Division of Genetic and Genomic Medicine and the Heart Center Nationwide Children's Hospital, Department of Pediatrics College of Medicine Ohio State University  Columbus Ohio USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hans Tomas","family":"Bjornsson","sequence":"additional","affiliation":[{"name":"Landspitali University Hospital  Reykjavik Iceland"},{"name":"Faculty of Medicine University of Iceland  Reykjavik Iceland"},{"name":"Mckusick\u2010Nathans Department of Genetic Medicine Johns Hopkins University  Baltimore Maryland USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Brynja Bjork","family":"Gunnarsdottir","sequence":"additional","affiliation":[{"name":"Landspitali University Hospital  Reykjavik Iceland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Amy","family":"Goldstein","sequence":"additional","affiliation":[{"name":"Children's Hospital of Philadelphia  Philadelphia Pennsylvania USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Scott A.","family":"Falk","sequence":"additional","affiliation":[{"name":"Perelman School of Medicine of the University of Pennsylvania  Philadelphia Pennsylvania USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2022,9,21]]},"reference":[{"key":"e_1_2_11_2_1","unstructured":"US Food and Drug Administration.Expanded access to investigational drugs for treatment use\u2014questions and answers. 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