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Article

Pathophysiology of propionic and methylmalonic acidemias. Part 1: Complications

23 May 2019

Abstract excerpt

Over the last decades, advances in clinical care for patients suffering from propionic acidemia (PA) and isolated methylmalonic acidemia (MMA) have resulted in improved survival. These advances were possible thanks to new pathophysiological insights. However, patients may still suffer from devastating complications which largely determine the unsatisfying overall outcome. To optimize our treatment strategies,...

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PMID: 31119747
DOI: 10.1002/jimd.12129

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