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.
Mitochondrial Disease Workshop Shows US FDA Commitment To First Approvals
First FDA-hosted workshop on rare family of mitochondrial diseases offers plenty of opportunities to talk challenges in R&D – and identify some solutions.
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