Diaz GA, Giugliani R, Guffon N, Jones SA, Mengel E et al.
Long-term safety and clinical outcomes of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency: two-year results.
Orphanet J Rare Dis. 2022;17:437
Wasserstein M, Lachmann R, Hollak C, Arash-Kaps L, Barbato A et al.
A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic acid sphingomyelinase deficiency (ASMD) in adults: One-year results.
Genet Med. 2022 ;24:1425-1436
Wasserstein M, Dionisi-Vici C, Giugliani R, Hwu WL, Lidove O, Lukacs Z, Mengel E, Mistry PK, Schuchman EH, McGovern M. Recommendations for clinical monitoring of patients with acid sphingomyelinase deficiency (ASMD).
Mol Genet Metab. 2019;126:98-105