Survival achieved in infants with rapidly progressive LAL-D via sebelipase alfa ERT: Results from the International LAL-D Registry von Vijay, Suresh, Evans, Jennifer, Jones, Simon, Lacaille, Florence, Abel, Florian, Montero, Javier Adolfo De Las Heras

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Survival achieved in infants with rapidly progressive LAL-D via sebelipase alfa ERT: Results from the International LAL-D Registry von Vijay, Suresh, Lacaille, Florence, de las Heras Montero, Javier Adolfo, Abel, Florian, Evans, Jennifer, Jones, Simon

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Successful desensitization protocol to alglucosidase and avalglucosidase alfa in a patient with infantile-onset Pompe disease von Montero, Javier Adolfo de las Heras, Gendive, Miriam, Sordo, Beatriz, Arza, Arantza, Segurola, Aritza, Unceta, María, Delgado, Teresa Cardoso

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Safety and pharmacokinetics of eliglustat administered with and without imiglucerase in pediatric participants with Gaucher disease type 1 and type 3: The ELIKIDS study von Gokcay, Gulden, Raiman, Julian A., Kissell, Julie, Cherkasov, Dmitry, Li, Ning, Xu, Christine, Xia, Binfeng, Cil, Sefika Uslu, de las Heras Montero, Javier Adolfo, Batsu, Isabela

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

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