Sebelipase alfa for lysosomal acid lipase deficiency: 5‐year treatment experience from a phase 2 open‐label extension study

BACKGROUND AND AIMS: Lysosomal acid lipase deficiency is characterized by hepatomegaly and dyslipidaemia, which can lead to cirrhosis and premature atherosclerosis. Sebelipase alfa is an approved recombinant human lysosomal acid lipase. In an open‐label extension study of adults with lysosomal acid...

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Vydáno v:Liver Int
Hlavní autoři: Malinová, Vĕra, Balwani, Manisha, Sharma, Reena, Arnoux, Jean‐Baptiste, Kane, John, Whitley, Chester B., Marulkar, Sachin, Abel, Florian
Médium: Artigo
Jazyk:Inglês
Vydáno: John Wiley and Sons Inc. 2020
Témata:
Genetics and Rare Liver Diseases
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7540377/
https://ncbi.nlm.nih.gov/pubmed/32657505
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/liv.14603
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