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TrialGenetics in medicine : official journal of the American College of Medical Genetics2021

One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency.

George A Diaz et al.PubMed ↗Full text ↗Publisher ↗

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The trial

Full record →Abstract, authors, funding and every citing paper · PMID 33875845