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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Dades bibliogràfiques
Publicat a:Liver Int
Autors principals: Malinová, Vĕra, Balwani, Manisha, Sharma, Reena, Arnoux, Jean‐Baptiste, Kane, John, Whitley, Chester B., Marulkar, Sachin, Abel, Florian
Format: Artigo
Idioma:Inglês
Publicat: John Wiley and Sons Inc. 2020
Matèries:
Genetics and Rare Liver Diseases
Accés en línia: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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