학술논문
Adults with chronic acid sphingomyelinase deficiency show significant visceral, pulmonary, and hematologic improvements after enzyme replacement therapy with olipudase-alfa: 1-year results of the ASCEND placebo-controlled trial.
Document Type
Article
Author
Wasserstein, Melissa; Arash-Kaps, Laila; Barbato, Antonio; Gallagher, Renata C.; Giugliani, Roberto; Guelbert, Norberto B.; Hollak, Carla; Ikezoe, Takayuki; Lachmann, Robin; Lidove, Oliver; Mabe, Paulina; Mengel, Eugen; Scarpa, Maurizio; Senates, Ebubekir; Tchan, Michel; Villarrubia, Jesus; Chen, Yixin; Fraser, Patricia A.; Thurberg, Beth L.; Zaher, Atef
Source
Subject
*NIEMANN-Pick diseases
*ENZYMES
*ADULTS
*PLATELET count
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Language
ISSN
1096-7192