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)