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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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Detalles Bibliográficos
Autores principales:	Malinová, Vĕra, Balwani, Manisha, Sharma, Reena, Arnoux, Jean‐Baptiste, Kane, John, Whitley, Chester B., Marulkar, Sachin, Abel, Florian
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2020
Materias:	Genetics and Rare Liver Diseases
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7540377/ https://www.ncbi.nlm.nih.gov/pubmed/32657505 http://dx.doi.org/10.1111/liv.14603

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