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MADEO , Annalisa

Last name: MADEO

First name: Annalisa

2018 (v1)

Publication

Metadata-only

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

Di Rocco, Maja Pisciotta, Livia Madeo, Annalisa

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 lysosomal disease. Ezetimibe is an azetidine...

Uploaded on: April 14, 2023