
Using AI and Longitudinal Data to Transform Rare Disease Care
May 7, 2026 - 00:19:21
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Families of children with the ultra-rare mitochondrial disorder pyruvate dehydrogenase complex deficiency, or PDCD, are fighting to get the U.S. Food and Drug Administration to provide a path to approval Saol Therapeutic...
When Endpoints Miss the Point is an episode from RARECast by RARECast. Families of children with the ultra-rare mitochondrial disorder pyruvate dehydrogenase complex deficiency, or PDCD, are fighting to get the U.S. Food and Drug Administra...
This episode belongs to RARECast.
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Published Mar 12, 2026, 00:36:47 long, audio available.