Navegação por Autor "Qi, Zhang"

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One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency

Diaz, George A.; Jones, Simon A.; Scarpa, Maurizio; Mengel, Karl Eugen; Giugliani, Roberto; Guffon, Nathalie; Batsu, Isabela; Fraser, Patricia A.; Jing, Li; Qi, Zhang; Renon, Catherine Ortemann (2021) [Artigo de periódico]

PURPOSE: To assess olipudase alfa enzyme replacement therapy for non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in children. METHODS: This phase 1/2, international, multicenter, ...