ArticleEuropean heart journal. Case reports2025
Expanded-access use of elamipretide in a newborn with Barth syndrome: a case report.
Article in European heart journal. Case reports, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
1 citing paper in PubMed.
- Elamipretide in pediatric Barth syndrome: from heart failure to school return.Orphanet journal of rare diseases · 2026Article
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3 authors.
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Abstract
Background: Barth syndrome (BTHS) is a rare genetic disease, with no approved curative therapies, characterized by abnormally developed cardiolipin, resulting in mitochondrial dysfunction. Cardiomyopathy, a common clinical manifestation of BTHS, often appears in infancy. Elamipretide, an investigational drug that binds to cardiolipin on the inner mitochondrial membrane, leads to improved membrane stability, enhanced adenosine triphosphate production, and reduced reactive oxygen species. This patient case aims to further support elamipretide's role in treating BTHS infants. Case summary: We present an infant diagnosed Discussion: Our case suggests that elamipretide may have contributed to the improvement of LV function in this BTHS infant, supporting elamipretide's early use in BTHS. Our findings align with the previous studies in which elamipretide treatment demonstrated normalization of mitochondrial function and improvement in LV function.
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