Long-Term Outcome of Infantile Onset Pompe Disease Patients Treated with Enzyme Replacement Therapy - Data from a German-Austrian Cohort.

Charlotte Pfrimmer,Martin Smitka,Nicole Muschol,Ralf A. Husain,Martina Huemer,Julia B. Hennermann,Rahel Schuler,Andreas Hahn

JOURNAL OF NEUROMUSCULAR DISEASES（2024）

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Infantile onset Pompe disease,prognosis,long-term outcome

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