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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Salvato in:
Dettagli Bibliografici
Pubblicato in:Liver Int
Autori principali: Malinová, Vĕra, Balwani, Manisha, Sharma, Reena, Arnoux, Jean‐Baptiste, Kane, John, Whitley, Chester B., Marulkar, Sachin, Abel, Florian
Natura: Artigo
Lingua:Inglês
Pubblicazione: John Wiley and Sons Inc. 2020
Soggetti:
Genetics and Rare Liver Diseases
Accesso online: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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