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Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months

Wasserstein, MP; Diaz, GA; Lachmann, RH; Jouvin, MH; Nandy, I; Ji, AJ; Puga, AC

Puga, AC (reprint author), Sanofi Genzyme, Clin Dev, Rare Dis, 1 Ave Pierre Brossolette, F-91385 Chilly Mazarin, France.

JOURNAL OF INHERITED METABOLIC DISEASE, 2018; 41 (5): 829

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