Acid sphingomyelinase deficiency: Burden of disease and real world impact of enzyme replacement therapy on pediatric patients and caregivers

Acid sphingomyelinase deficiency: Burden of disease and real world impact of enzyme replacement therapy on pediatric patients and caregivers

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Bibliographic Details
Published in:Molecular genetics and metabolism Vol. 138; no. 2; p. 107151
Main Authors: Hopkin, Justin, Donnelly, Conan, Crowe, Joslyn, Mathieson, Toni, Mbua, Solomon
Format: Journal Article
Language:English
Published: Elsevier Inc 01-02-2023
Online Access:Get full text
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Description
ISSN:1096-7192
1096-7206
DOI:10.1016/j.ymgme.2022.107151