Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study

Orphanet J Rare Dis. 2017 Feb 8;12(1):25.DOI:10.1186/s13023-017-0587-3

Jones SA, et al

https://www.ncbi.nlm.nih.gov/pubmed/28179030

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