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Distal Muscle Weakness is a Common and Early Feature in Long-Term Enzyme-Treated Classic Infantile Pompe Patients.

van den Dorpel J. J. A.,Poelman E.,Harlaar L.,van Kooten H. A.,van der Giessen L. J.,van Doorn P. A.,van der Ploeg A. T.,van den Hout J. M. P.,van der Beek N. A. M. E.

Orphanet journal of rare diseases（2020）

Cited 9|Views48
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Key words
Pompe disease,Glycogen storage disease type II,Enzyme replacement therapy (ERT),Muscle function,Distal muscle weakness
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