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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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Autors principals: 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
Format: Artigo
Idioma:Inglês
Publicat: 2013
Matèries:
Accés en línia: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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