P021: Long-term follow-up of cipaglucosidase alfa/miglustat in ambulatory and non-ambulatory patients with Pompe disease: An open-label phase I/II study (ATB200-02)

P021: Long-term follow-up of cipaglucosidase alfa/miglustat in ambulatory and non-ambulatory patients with Pompe disease: An open-label phase I/II study (ATB200-02)

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Bibliographic Details
Published in:Genetics in Medicine Open Vol. 1; no. 1; p. 100031
Main Authors: Kishnani, Priya, Schoser, Benedikt, Bratkovic, Drago, Clemens, Paula, Goker-Alpan, Ozlem, Ming, Xue, Roberts, Mark, Vorgerd, Matthias, Sivakumar, Kumaraswamy, van der Ploeg, Ans, Goldman, Mitchell, Wright, Jacquelyn, Holdbrook, Fred, Jain, Vipul, Das, Sheela Sitaraman, Wasfi, Yasmine, Mozaffar, Tahseen, Byrne, Barry
Format: Journal Article
Language:English
Published: Elsevier Inc 2023
Elsevier
Online Access:Get full text
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ISSN:2949-7744
2949-7744
DOI:10.1016/j.gimo.2023.100031