10.6084/m9.figshare.c.3686818_D1.v1 Simon Jones Simon Jones Sandra Rojas-Caro Sandra Rojas-Caro Anthony Quinn Anthony Quinn Mark Friedman Mark Friedman Sachin Marulkar Sachin Marulkar Fatih Ezgu Fatih Ezgu Osama Zaki Osama Zaki J. Gargus J. Gargus Joanne Hughes Joanne Hughes Dominique Plantaz Dominique Plantaz Roshni Vara Roshni Vara Stephen Eckert Stephen Eckert Jean-Baptiste Arnoux Jean-Baptiste Arnoux Anais Brassier Anais Brassier Kim-Hanh Le Quan Sang Kim-Hanh Le Quan Sang Vassili Valayannopoulos Vassili Valayannopoulos Additional file 1: Table S1. of Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study Springer Nature 2017 Lysosomal acid lipase deficiency Wolman disease Sebelipase alfa Survival Hemophagocytic lymphohistiocytosis 2017-02-08 05:00:00 Journal contribution https://springernature.figshare.com/articles/journal_contribution/Additional_file_1_Table_S1_of_Survival_in_infants_treated_with_sebelipase_Alfa_for_lysosomal_acid_lipase_deficiency_an_open-label_multicenter_dose-escalation_study/4634554 Summary of dosing, dose changes, and current clinical status (patients who survived to age ≥24 months). Table S2. LAL enzyme activity and LIPA genotypes. Table S3. Changes in serum transaminases, total bilirubin, and γ-glutamyltransferase (full analysis set). Table S4. Proportion of patients meeting criteria for undernutrition in the primary efficacy analysis (patients who survived to age 12 months). Table S5. Serum transaminases, hemoglobin, and albumin, most recent measurement (patients who survived to age ≥24 months). Table S6. Treatment-emergent adverse events (TEAEs) reported for two or more patients in the full analysis set. Table S7. Summary of infusion-associated reactions (IARs) in the full analysis set. (DOCX 94 kb)