Source link : https://www.newshealth.biz/health-news/fda-reviewers-cast-doubt-on-drug-candidate-for-barth-syndrome/
Ahead of an FDA advisory committee meeting on Thursday, agency reviewers cast doubt on the efficacy of elamipretide for Barth syndrome, an ultra-rare disease that has no approved treatments. In its new drug application, Stealth BioTherapeutics described elamipretide as a first-in-class mitochondrial protective agent that theoretically improves the function of cardiolipin-deficient mitochondria in patients with […]
Author : News Health
Publish date : 2024-10-09 20:14:03
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