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Cipaglucosidase alfa plus miglustat in Pompe disease: two non-ambulatory patients switching from high‑dose, high-frequency alglucosidase alfa

Author:

Barry J. Byrne,Jeff Castelli,Vipul Jain,Sheela Sitaraman Das,Jennifer Zhang

Publication:

Neuromuscular Disorders

Publisher:

Elsevier

Date:

November 2025

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