A US Food and Drug Administration scientific workshop on mitochondrial diseases signaled the agency’s continued interest in helping drug developers get to the first approvals in disorders like Barth syndrome and Friedreich’s ataxia, but also underscored the continued challenges in designing clinical trials in such a genetically heterogeneous group of diseases.
In one sign of the agency’s willingness to encourage mitochondrial disorder drug development, FDA Office of Pediatric Therapeutics pediatric ethicist...
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