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TrialOrphanet journal of rare diseases2021

Long-term effectiveness of carglumic acid in patients with propionic acidemia (PA) and methylmalonic acidemia (MMA): a randomized clinical trial.

Majid Alfadhel et al.PubMed ↗Full text ↗Publisher ↗

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The trial

Full record →Abstract, authors, funding and every citing paper · PMID 34635114