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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Detalhes bibliográficos
Publicado no:Liver Int
Main Authors: Malinová, Vĕra, Balwani, Manisha, Sharma, Reena, Arnoux, Jean‐Baptiste, Kane, John, Whitley, Chester B., Marulkar, Sachin, Abel, Florian
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2020
Assuntos:
Genetics and Rare Liver Diseases
Acesso em linha: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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