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

Genetics in Medicine, Published online: 19 April 2021; doi:10.1038/s41436-021-01156-3One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency

Source: Genetics in Medicine - April 19, 2021 Category: Genetics & Stem Cells Authors: George A. Diaz Simon A. Jones Maurizio Scarpa Karl Eugen Mengel Roberto Giugliani Nathalie Guffon Isabela Batsu Patricia A. Fraser Jing Li Qi Zhang Catherine Ortemann-Renon Source Type: research