Long term substrate reduction therapy with ezetimibe alone or associated with statins in three adult patients with lysosomal acid lipase deficiency

BACKGROUND: Lysosomal acid lipase deficiency is an autosomal recessive metabolic disease with a wide range of severity from Wolman Disease to Cholesterol Ester Storage Disease. Recently enzyme replacement therapy with sebelipase alpha has been approved by drug agencies for treatment of this lysosoma...

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Detalles Bibliográficos
Autores principales: Di Rocco, Maja, Pisciotta, Livia, Madeo, Annalisa, Bertamino, Marta, Bertolini, Stefano
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5787265/
https://www.ncbi.nlm.nih.gov/pubmed/29374495
http://dx.doi.org/10.1186/s13023-018-0768-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5787265/
https://www.ncbi.nlm.nih.gov/pubmed/29374495
http://dx.doi.org/10.1186/s13023-018-0768-8

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