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Barth Syndrome - Springer Nature

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Last Updated: 10 September 2022

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Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome

The best squares mean difference between groups was 96. 7 m at week 64 and 91. 0 m at week 76 in favour of elamipretide. Left ventricular stroke volume increased from baseline in patients with elamipretide but not so low in NHCs. Conclusions Overall, the research found a NHC for use in determining the effectiveness of therapeutic interventions in patients with BTHS, and the results indicate that elamipretide may prolong natural heart function and functional capacity in patients with BTHS in comparison to NHC.

Source link: https://doi.org/10.1186/s13023-022-02469-5


Elamipretide for Barth syndrome cardiomyopathy: gradual rebuilding of a failed power grid

Patients with Barth syndrome have a high risk of mortality in infanthood and cardiomyopathy, as a result of significant immune system deficion. Results from several studies confirming the use of elamipretide in multiple models of disease are particularly encouraging, particularly in areas where there is a reported diagnosis of cardiomyopathy. This review explores the challenges and opportunities for treating Barth syndrome cardiomyopathy patients with elamipretide, as well as its potential to structurally alter the failing left ventricle at the global, cellular, and molecular level in a progressive manner by precise targeting of the mitochondrial inner membrane.

Source link: https://doi.org/10.1007/s10741-021-10177-8

* Please keep in mind that all text is summarized by machine, we do not bear any responsibility, and you should always check original source before taking any actions

* Please keep in mind that all text is summarized by machine, we do not bear any responsibility, and you should always check original source before taking any actions