Sebelipase alfa enzyme replacement therapy in Wolman disease: a nationwide cohort with up to ten years of follow-up.

Orphanet J Rare Dis · 2021 · PMC8670257 · PMID 34906190

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showed a trendno p-value reported

Under sebelipase alfa treatment, alanine transaminase, aspartate transaminase, gamma-glutamyltranspeptidase (γGT), total bilirubin and albumin showed a trend to improvement in all patients (Fig. 1 ).

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