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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

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journal contribution
posted on 08.02.2017, 05:00 by Simon Jones, Sandra Rojas-Caro, Anthony Quinn, Mark Friedman, Sachin Marulkar, Fatih Ezgu, Osama Zaki, J. Gargus, Joanne Hughes, Dominique Plantaz, Roshni Vara, Stephen Eckert, Jean-Baptiste Arnoux, Anais Brassier, Kim-Hanh Le Quan Sang, Vassili Valayannopoulos
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)