These authors contributed equally to this work.
Steatohepatitis/Metabolic Liver Disease
Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients With cholesteryl ester storage disease
Version of Record online: 28 MAR 2013
Copyright © 2013 American Association for the Study of Liver Diseases
Volume 58, Issue 3, pages 950–957, September 2013
How to Cite
Balwani, M., Breen, C., Enns, G. M., Deegan, P. B., Honzík, T., Jones, S., Kane, J. P., Malinova, V., Sharma, R., Stock, E. O., Valayannopoulos, V., Wraith, J. E., Burg, J., Eckert, S., Schneider, E. and Quinn, A. G. (2013), Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients With cholesteryl ester storage disease. Hepatology, 58: 950–957. doi: 10.1002/hep.26289
Potential conflict of interest: Synageva Biopharma Corp. is the manufacturer of sebelipase alfa and conducted the statistical analysis. All authors had access to the study data, reviewed early and final drafts of the article, and are fully responsible for the content and editorial decisions related to the article. Drs. Burg, Quinn, and Schneider own stocks in and are employees of Synageva BioPharma. Dr. Deegan received grants from Synageva BioPharma. Dr. Eckert is an employee of Synageva BioPharma. Dr. Jones consults for and advises Synageva BioPharma. Dr. Wraith received grants from Synageva BioPharma, Genozyme, BioMarin, and Shire.
See Editorial on Page 850
Supported by Synageva BioPharma Corp. and in part by grant UL1TR000067 from the National Center for Advancing Translational Sciences, National Institutes of Health to the Mount Sinai School of Medicine and by research project PRVOUK-P24/LF1/3 to TH.
- Issue online: 29 AUG 2013
- Version of Record online: 28 MAR 2013
- Accepted manuscript online: 24 JAN 2013 10:00AM EST
- Manuscript Accepted: 3 JAN 2013
- Manuscript Received: 20 AUG 2012
Additional Supporting Information may be found in the online version of this article.
|hep26289-sup-0001-suppfig1.tiff||3478K||Supporting Information Figure 1. Mean hepatic transaminases, alkaline phosphatase, and gamma-glutamyl transpeptidase (U/L) in 7 patients in the LAL-CL01 and LAL-CL04 study.|
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