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NEO1 and NEO-EXT Studies: Long-term Safety of Repeat Avalglucosidase Alfa Dosing for 4.5 Years in Late-Onset Pompe Disease Patients

Loren Pena,Richard J. Barohn,Mazen M. Dimachkie,Priya S. Kishnani,Shafeeq Ladha,Karl-Eugen Mengel,Sabrina Sacconi,Philip Van Damme,John Vissing,Kristina An Haack,Christopher Hug,Judith Johnson,Charlotte Sensinger,Benedikt Schoser

Molecular Genetics and Metabolism（2019）

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