Article

Use of Elamipretide in Patients Assigned Treatment in the Compassionate Use Program: Case Series in Four Rare Orphan Diseases

2021-10-12

Abstract excerpt

<title>Abstract</title> <p><bold>Background:</bold>Several mitochondrial diseases are caused by pathogenic variants that impair membrane phospholipid remodeling. There are no US Food and Drug Administration (FDA)–approved therapies for these conditions. Elamipretide is a cell-permeable peptide that has been shown to target the inner mitochondrial membrane where it binds to cardiolipin, resulting in improved membr...

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Literature Corpus work: 6b1119c3-8ff0-52c1-9f6f-6e12bed565e3
DOI: 10.21203/rs.3.rs-927266/v1

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