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Volume 61, Issue 5, Pages (November 2014)

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1 Volume 61, Issue 5, Pages 1135-1142 (November 2014)
Sebelipase alfa over 52weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency  Vassili Valayannopoulos, Vera Malinova, Tomas Honzík, Manisha Balwani, Catherine Breen, Patrick B. Deegan, Gregory M. Enns, Simon A. Jones, John P. Kane, Eveline O. Stock, Radhika Tripuraneni, Stephen Eckert, Eugene Schneider, Gavin Hamilton, Michael S. Middleton, Claude Sirlin, Bruce Kessler, Christopher Bourdon, Simeon A. Boyadjiev, Reena Sharma, Chris Twelves, Chester B. Whitley, Anthony G. Quinn  Journal of Hepatology  Volume 61, Issue 5, Pages (November 2014) DOI: /j.jhep Copyright © 2014 European Association for the Study of the Liver Terms and Conditions

2 Fig. 1 Flow chart diagram of the LAL-CL01 and LAL-CL04 study design. Subjects (18–65yr) were required to have documented LAL deficiency, hepatomegaly and/or transaminases >1.5×ULN, and may be on a lipid-lowering agents (stable dose for ⩾4weeks). LAL, Lysosomal acid lipase; qw, once weekly; qow, once every other week. Journal of Hepatology  , DOI: ( /j.jhep ) Copyright © 2014 European Association for the Study of the Liver Terms and Conditions

3 Fig. 2 Mean hepatic transaminases (U/L) in LAL-CL04 study (n=8). All one-week-post-infusion laboratory data are presented one week after the week 24 visit. ALT, alanine transaminase; AST, aspartate aminotransferase; qw, once weekly; qow, once every other week. Journal of Hepatology  , DOI: ( /j.jhep ) Copyright © 2014 European Association for the Study of the Liver Terms and Conditions

4 Fig. 3 Mean percent change from LAL-CL01 baseline for serum lipids in LAL-CL04 study (n=8). All one-week-post-infusion laboratory data are presented one week after the week 24 visit. HDL, high-density lipoprotein-cholesterol; LDL, low density lipoprotein-cholesterol; Trig, triglycerides. Journal of Hepatology  , DOI: ( /j.jhep ) Copyright © 2014 European Association for the Study of the Liver Terms and Conditions

5 Fig. 4 Absolute LDL values over time (n=8).
Journal of Hepatology  , DOI: ( /j.jhep ) Copyright © 2014 European Association for the Study of the Liver Terms and Conditions

6 Fig. 5 Change in liver volume and hepatic PDFF in LAL-CL04. ∗One patient did not have a LAL-CL04 baseline assessment, thus, numbers are decreased by one, relative to liver volume. Journal of Hepatology  , DOI: ( /j.jhep ) Copyright © 2014 European Association for the Study of the Liver Terms and Conditions


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