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These symptoms typically improve after discontinuation

This phenocopied the decrease in glucose-derived acetyl-CoA pools following knockdown of the de novo carnitine-synthesis enzyme TMLHE (Extended Data Fig

Persistent vomiting that prevents you from keeping down fluids or food is a red flag that demands immediate medical attention, as dehydration can compound metabolic complications

Earlier phase 2 trials tested different timing protocols, including 2 week escalations and faster progressions to higher doses