L-carnitine Supplements May Help Augment Enzyme Replacement Therapy

L-carnitine Supplements May Help Augment Enzyme Replacement Therapy

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L-carnitine Supplements May Help Augment Enzyme Replacement Therapy

L-carnitine — a molecule often taken as an oral dietary supplement — boosts the efficacy of enzyme replacement therapy (ERT), increasing by four times the activity of acid alpha-glucosidase (GAA), the faulty enzyme in Pompe disease, in patient-derived cells, a study shows. The molecule was found to stabilize GAA, working as a potential chaperone therapy but without the suppressing effects of those currently in development for Pompe. These findings suggest that adding L-carnitine to standard ERT…

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