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. 2024 Jun 22:2:101859.
doi: 10.1016/j.gimo.2024.101859. eCollection 2024.

Expanded-access use of elamipretide in a critically ill patient with Barth syndrome

Amy C Goldstein  1   2 , Cassandra Pantano  1   2 , Mariya Redko  1 , Laura E MacMullen  1 , Katsuhide Maeda  2   3 , Matthew J O'Connor  2   4
Affiliations

Expanded-access use of elamipretide in a critically ill patient with Barth syndrome

Amy C Goldstein et al. Genet Med Open. .
No abstract available

Keywords: Barth syndrome; Cardiolipin; Cardiomyopathy; Elamipretide; Mitochondria.

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Conflict of interest statement

The authors declare no conflicts of interest.

Figures

Figure 1
Figure 1
Left ventricular ejection fraction over time during elamipretide treatment.
Figure 2
Figure 2
Proposed disease pathway of Barth syndrome. ATP, adenosine triphosphate; ROS, reactive oxygen species.
See this image and copyright information in PMC

References

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    1. Expanded access to investigational drugs for treatment use, questions and answers, Guidance for industry. Draft guidance. United States Food and Drug Administration. https://www.fda.gov/media/162793/download
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    1. Sabbah H.N., Taylor C., Vernon H.J. Temporal evolution of the heart failure phenotype in Barth syndrome and treatment with elamipretide. Future Cardiol. 2023;19(4):211–225. doi: 10.2217/fca-2023-0008. - DOI - PubMed
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