Higher dose alglucosidase alfa is associated with improved overall survival in infantile-onset Pompe disease (IOPD): data from the Pompe Registry.

Autor: Kishnani, Priya S.1 (AUTHOR) priya.kishnani@duke.edu, Kronn, David2 (AUTHOR), Suwazono, Shugo3 (AUTHOR), Broomfield, Alexander4 (AUTHOR), Llerena, Juan5 (AUTHOR), Al-Hassnan, Zuhair Nasser6 (AUTHOR), Batista, Julie L.7 (AUTHOR), Wilson, Kathryn M.8 (AUTHOR), Periquet, Magali9 (AUTHOR), Daba, Nadia10 (AUTHOR), Hahn, Andreas11 (AUTHOR), Chien, Yin-Hsiu12 (AUTHOR)
Zdroj: Orphanet Journal of Rare Diseases. 12/6/2023, Vol. 18 Issue 1, p1-13. 13p.
Databáze: Academic Search Ultimate
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