TWO-YEAR OUTCOMES OF OLIPUDASE ALFA THERAPY IN CHILDREN WITH CHRONIC ACID SPHINGMYELINASE DEFICIENCY

Diaz, G; Giugliani, R; Guffon, N; Jones, S; Mengel, E; Scarpa, M; Witters, P; Yarramaneni, A; Rawlings, A; Kim, Y; Kumar, M

PEDIATRIC BLOOD & CANCER, 2022; 69 ():

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