Novel treatment options for lysosomal acid lipase deficiency: critical appraisal of sebelipase alfa

Lysosomal acid lipase deficiency (LAL-D) is a rare disorder of cholesterol metabolism with an autosomal recessive mode of inheritance. The absence or deficiency of the LAL enzyme gives rise to pathological accumulation of cholesterol esters in various tissues. A severe LAL-D phenotype manifesting in...

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Detalles Bibliográficos
Autores principales: Su, Kim, Donaldson, Emma, Sharma, Reena
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Dove Medical Press 2016
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5074735/
https://www.ncbi.nlm.nih.gov/pubmed/27799810
http://dx.doi.org/10.2147/TACG.S86760

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5074735/
https://www.ncbi.nlm.nih.gov/pubmed/27799810
http://dx.doi.org/10.2147/TACG.S86760

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