Marklald: Plasma biomarkers as key indicators for monitoring patients with lysosomal acid lipase deficiency treated with sebelipase alfa. Preliminary findings from a multicentre cohort study in Spain

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Published in:Molecular genetics and metabolism 2025-02, Vol.144 (2), p.108916, Article 108916
Main Authors: Roca-Esteve, Sonia, Gonzalo, Irene Serrano, Montero, Javier Adolfo De Las Heras, Bernabeu, Jesus Quintero, Mercadal-Hally, Maria, Almohalla, Carolina, Balmaseda, Elena, Blasco-Alonso, Javier, Martínez, Marta Casañas, Lopez, Maria Jose De Castro, Madurga, Laura Escartín, Gutiérrez Carrasquilla, Liliana P., Berto, Tomas Hernández, Ibarretxe, Daiana, Blázquez, Cristina Iglesias, Martinez-Triguero, Maria Luisa, Mohand, Patricia Pérez, Gil, Nuria P., Ramos, Andrea S., Yahyaoui, Raquel, de Frutos, Laura López, Giraldo, Pilar, Cebolla, Jorge J.
Format: Article
Language:English
Online Access:Get full text
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ISSN:1096-7192
DOI:10.1016/j.ymgme.2024.108916