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

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 seb...

Source: Orphanet Journal of Rare Diseases - January 27, 2018 Category: Internal Medicine Authors: Maja Di Rocco, Livia Pisciotta, Annalisa Madeo, Marta Bertamino and Stefano Bertolini Tags: Research Source Type: research