Listar por autor "Yong, Kim"
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Long-term safety and clinical outcomes of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency : two-year results
Diaz, George A.; Giugliani, Roberto; Guffon, Nathalie; Jones, Simon A.; Mengel, Eugen; Scarpa, Maurizio; Witters, Peter; Yarramanen, Abhimanyu; Jing, Li; Armstrong, Nicole M.; Yong, Kim; Renon, Catherine Ortemann; Kumar, Monica (2022) [Artículo de periódico]Background Acid sphingomyelinase deficiency (ASMD) is a lysosomal disorder caused by deficiency of acid sphingomyelinase (ASM) leading to the accumulation of sphingomyelin (SM) in a variety of cell types. Lysosphingomyelin ...