This suggests that cardiolipin metabolism may be a common pathway in various mitochondrial-related diseases, including Barth syndrome[4]. Finally, a new murine model of Barth syndrome has been ...
The US Food and Drug Administration (FDA) has delayed its decision on Stealth BioTherapeutics’ new drug application (NDA) for elamipretide, a treatment for the ultra-rare disease Barth syndrome.
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