Clinical Effect and Safety Profile of Recombinant Human Lysosomal Acid Lipase in Patients with Cholesteryl Ester Storage Disease

BACKGROUND & AIMS: Cholesteryl Ester Storage Disease, an inherited deficiency of lysosomal acid lipase, is an underappreciated cause of progressive liver disease with no approved therapy. Presenting features include dyslipidemia, elevated transaminases, and hepatomegaly. METHODS: To assess the c...

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Detaylı Bibliyografya
Asıl Yazarlar: Balwani, Manisha, Breen, Catherine, Enns, Gregory M, Deegan, Patrick B, Honzík, Tomas, Jones, Simon, Kane, John P, Malinova, Vera, Sharma, Reena, Stock, Eveline O, Valayannopoulos, Vassili, Wraith, J Edmond, Burg, Jennifer, Eckert, Stephen, Schneider, Eugene, Quinn, Anthony G
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: 2013
Konular:
Article
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC3728169/
https://ncbi.nlm.nih.gov/pubmed/23348766
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/hep.26289
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