Enzyme replacement therapy in lysosomal acid lipase deficiency (LAL-D): a systematic literature review.

Aamir BashirPramil TiwariAjay Duseja

Published in: Therapeutic advances in rare disease (2021)

= 0.08 respectively. Mostly the adverse events related to the infusions were infrequent and mild-to-moderate in severity. The enzyme replacement provides an effective, safe and well tolerated treatment in both variants of LAL-D.

Keyphrases

replacement therapy
smoking cessation
copy number
gene expression
dna methylation